Pipeline of GD & GBA-PD

This generated page lists source notes whose first nav_path entry is Pipeline of GD & GBA-PD. It is a coverage index, not a full synthesis page.

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20240722_181741Pipeline of GD & GBA-PDPipeline of GD & GBA-PD; NLRP3 PDE; 이주경; ADPD 2024; GCB PET / Lysosomotropic PET Notes; …
20240722_181748Pipeline of GD & GBA-PD > SummaryPipeline of GD & GBA-PD; Two Group Proportion Comparison; GBA-PD / sPD / HC Biomarker Matrix; Cognitive Readouts; Summary; …
20240722_181752Pipeline of GD & GBA-PD > SummaryPipeline of GD & GBA-PD; Eliglustat; Ambroxol; Mechanism; Preclinical Of Ambroxol; …
20240722_181756Pipeline of GD & GBA-PD > Venglustat (Ibiglustat)Pipeline of GD & GBA-PD; Ambroxol; Clinical Trials Of Ambroxol; Venglustat (Ibiglustat); Preclinical; …
20240722_181800Pipeline of GD & GBA-PD > Prevail PR001Pipeline of GD & GBA-PD; Venglustat (Ibiglustat); Clinical Trials Of Venglustat; LEAP Biomarker Notes; MOVES-PD Biomarker And Clinical Results; …
20240722_181805Pipeline of GD & GBA-PD > Prevail PR001Pipeline of GD & GBA-PD; Prevail PR001; Preclinical; PR001 Single-Dose Efficacy Study In The Genetic Mouse Model; Biodistribution And GCase Activity; …
20240722_181809Pipeline of GD & GBA-PD > Prevail PR001Pipeline of GD & GBA-PD; PR001 Mouse Data: 4L/PS-NA Genetic Mouse Model; PR001 Mouse Data: A53T + CBE Model; Dose-Finding NHP ICM Biodistribution; Vector Genome Heatmap; …
20240722_181813Pipeline of GD & GBA-PD > SummaryPipeline of GD & GBA-PD; PR001A / PROPEL: Biomarker Goal And Interim Result; PR001A / PROVIDE; PR001A / PROGRESS; PR001A / PROCEED; …
20240722_181818Pipeline of GD & GBA-PD > TAK-341 (MEDI1341) > PK/PD modelPipeline of GD & GBA-PD; TAK-341 (MEDI1341); MEDI1341 Effector Function Note; MEDI1341 Affinity; Figure 4; …
20240722_182123Pipeline of GD & GBA-PD > Summary총정리 GBA protein & activity POSTMORTEM (continued); Synapse; 총정리 GBA protein & activity POSTMORTEM] Activity: hetero only 에선 kurzawa가 유일.; PD only / PD+ heteroz / Hetero only / GD only / PD+GD matrix; Peripheral blood and GD studies; …
20240722_182126Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity총정리 GBA protein & activity (continued); Uncertain Spans
20240722_182130Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity총정리 GBA protein & activity (continued); Uncertain Spans
20240722_182133Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity총정리 GBA protein & activity (continued); Uncertain Spans
20240722_182136Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity > Summary GBA protein & activity, GlcCer, GlcSphMarker priority, 202107 Comp lysosomal target dashboard, and Summary GBA protein & activity, GlcCer, GlcSph; Marker priority; 202107 Comp lysosomal target dashboard; Summary GBA protein & activity, GlcCer, GlcSph; Uncertain Spans
20240722_182139Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity > Summary GBA protein & activitySummary GBA protein & activity (continued), Summary table: GBA-PD patients, and Read the Paper (A) GBA Pathway Biomarkers; Summary table: GBA-PD patients; Read the Paper (A) (GBA Pathway Biomarkers for Genetic and Idiopathic PD); Uncertain Spans
20240722_182143Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity > Read the PaperRead the Paper (>) GBA Pathway Biomarkers for Genetic and Idiopathic PD; Stratification by GBA pathway; 총정리중 GBA+PD에서 Lysosomal 반; S: Lysosome Lysosomal autophagy POSTMORTEM; Lysosomal autophagy model rows; …
20240722_182146Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity총정리 GBA protein & activity (continued), Sidransky 2012 review, Saposin C summary; [Gegg 2012] GBA activity / protein level; Table Below by Sidransky 2012 review; Saposin C summary; Lower Saposin / GBA table; …
20240722_182149Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity > a-syn POSTMORTEMa-syn POSTMORTEM, a-syn SH-SY5Y + Cullen, a-syn PC only, FRACTIONATION; Choi 2011 요약; Postmortem rows (Mazzulli 2011, Murphy 2013, Murphy 2014); a-syn SH-SY5Y + Cullen: probably this is all for cell line]; a-syn PC only; …
20240722_182153Pipeline of GD & GBA-PD > Summary > GBASidransky 2012 review (continued), PD-GD / GBA1-PD notes, GBA gene, GBA Mutation detection method; Sidransky 2012 review table continuation; PD-GD / GBA1-PD notes; GBA; GBA Mutation detection method; …
20240722_182157Pipeline of GD & GBA-PD > GBA > Structure of GBA proteinCarrier screening (continued), Structure / Function / Turnover / Expression / Lifecycle of GBA, activity (‘BGL test’); Carrier screening (continued); Structure of GBA protein (EC 3.2.1.45); Function of GBA protein; Turnorver of GBA protein; …
20240722_182200Pipeline of GD & GBA-PD > GBA > GBA activity (BGL test)GBA activity (‘BGL test’); Product (kit); Uncertain Spans
20240722_182203Pipeline of GD & GBA-PD > GBA > Catalytic pathwaySebastian table tail, Product (kit), Patient selection, Catalytic pathway, GSL flux, GluCer / GalCer isoforms, Gangliosides; Sebastian 20201124 (continued); Product (kit); Catalytic pathway; GluCer (Glucosylceramide) isoforms; …
20240722_182207Pipeline of GD & GBA-PD > GBA > Catalytic pathway > GangliosidesGluCer / GalCer isoforms (continued), Gangliosides, Sphingolipid pathway; GlcCer (Glucosylceramide); Gangliosides; Salvage pathway / Degradation; Sphingolipid base structure; …
20240722_182210Pipeline of GD & GBA-PD > GBA > Catalytic pathway > GangliosidesGangliosides Synthesis / Salvage / Degradation, Sphingolipid base, Disease cascade, Glycolipid metabolism, GBA chaperone; Gangliosides table (continuing from previous capture); Salvage pathway / Degradation; Sphingolipid base structure; Ganglioside / sphingolipid degradation cascade (figure labels); …
20240722_182213Pipeline of GD & GBA-PD > GBA > Catalytic pathway > Gangliosides > GlcSphDisease cascade tail, Glycolipid metabolism, GBA1 / GlcSph cycle, GBA chaperone / GCS inhibitor, GlcSph section; Ganglioside / sphingolipid degradation cascade tail (figure labels); 위 그림에서 cerebroside 는 = GlcCer, GalCer; Glycolipid metabolism schematic; GBA1 / GlcSph cycle inset; …
20240722_182216Pipeline of GD & GBA-PD > GD > Mutations SummaryGlcSph (continued) / GD: Dx, Epidemiology, Mutations Summary, mutation distribution; GD; Dx of GD; Epidemiology of GD (prevalence); Mutations Summary; …
20240722_182219Pipeline of GD & GBA-PD > GD > Types and NeuropathologyPathophysiology In GD, GBA1 mutations, Event / Observational markers, Types and Neuropathology; Pathophysiology In GD; Types and Neuropathology; Uncertain Spans
20240722_182224Pipeline of GD & GBA-PD > GD > Parameters > GD 1 SummaryType 3 / GD3 (continued), CNS Sx in GD3, Parameters / GD 1 Summary; Type 3 (continued); Type 3 + PD; CNS Sx in GD3; Parameters / GD 1 Summary; …
20240722_182227Pipeline of GD & GBA-PD > GD > Parameters > GD 3 SummaryGD 1 Summary tail, GD 3 Summary, GlcCer / GlcSph (detail), Narita 2016 patient table; GD 1 Summary (continued); SRT response (Smid 2016 #529); 2019 Lukina (Eliglustat); GD 3 Summary; …
20240722_182230Pipeline of GD & GBA-PD > GD > Parameters > GD 3 SummaryNarita 2016 patient body, Plasma in GD 1, Reference values, Animal models of GD; Narita 2016 #560 (continued); Plasma in GD 1; Experimental; Tottori univ / Dried blood spot in GD 3; …
20240722_182233Pipeline of GD & GBA-PD > GD > Animal models of GD2019 Lukina (re-shown), Animal models of GD (continued), hGBA-L444P-tg8 mouse, GCB Mouse Model Substrate Comparison; 2019 Lukina Eliglustat (re-shown); Animal models of GD (continued); hGBA-L444P-tg8 figures and notes; GCB Mouse Model Substrate Comparison; …
20240722_182237Pipeline of GD & GBA-PD > GD > Animal models of GDD409V/D409V KI, GBA enzyme assay, conditional / nGD models, animal models comparison, Saposin C dose; D409V/D409V KI; GBA enzyme assay; MJFF: GBA D409VKi (hom) and crossed them with thy-1 aSyn or introduced PFF; nGD model / conditional; …
20240722_182241Pipeline of GD & GBA-PD > GD > Animal models of GD > Outcome measure in nGDStephens 1978 (continued), animal model literature rows, Outcome measure in nGD (SST / mSST); Stephens 1978 (continued); Animal model literature rows; Outcome measure in nGD; mSST domain scoring; …
20240722_182245Pipeline of GD & GBA-PD > GD > Parameters > Oculomotor measurementsOutcome measure (continued), Sample size in nGD, Oculomotor measurements, Saccade & PLR, Retina in nGD; Outcome measure (continued); Sample size estimation in nGD; Oculomotor measurements; Photoreceptors (in retina겠지); …
20240722_182248Pipeline of GD & GBA-PD > GD > Parameters > Retina in nGDHopf saccadometry tail, Retina in nGD / Retina & LSD, PLR / chromatic pupillometry, ERG, CRO / KOL, Lipofuscin; Hopf saccadometry (continued); Retina in nGD; Retina & LSD; PLR / chromatic pupillometry; …
20240722_182252Pipeline of GD & GBA-PD > GD > Parameters > Oculomotor measurementsRGC, Retinal PK, Short ciliary nerves, Antiepileptic drugs / retinal layers, RGC table, Study plan; RGC; Retinal PK; Short ciliary nerves; The effect of antiepileptic drugs on visual performance; …
20240722_182255Pipeline of GD & GBA-PD > GBA-PD > Mutation GBA-PD2021 WORLDSymposium Ayuko Iverson, Narita Aya, Handover to Otake, GBA-PD penetrance, Mutation GBA-PD; 2021 WORLDSymposium Ayuko Iverson; Narita Aya (Iottori / Tottori univ); Handover to Otake on GD; GBA-PD / Penetrance (LIFETIME); …
20240722_182259Pipeline of GD & GBA-PD > GBA-PD > MOA GBA-PDPostmortem tail, Odds ratios, Phenotype / Cognition GBA-PD, Gene Dose Effect, MOA GBA-PD; Postmortem GBA-PD (continued); Odds ratios; Phenotype of GBA-PD; Cognition GBA-PD; …
20240722_182303Pipeline of GD & GBA-PD > GBA-PD > Motor vs CognitionaSyn spreading tail, ER stress, OS, Inflammation, Motor vs Cognition, Cognition / Dementia; aSyn spreading (continued); ER stress; OS; Inflammation; …
20240722_182306Pipeline of GD & GBA-PD > GBA-PD > Animal models of GBA-PDIn vitro GBA-PD tail, 20191125 TAK Okai matrix, 20201209_ACi overview, Animal models of GBA-PD start; In vitro GBA-PD (continued); Below from 20191125 TAK Okai san slide gba-gt slide; Below from 20201209_ACi overview; Animal models of GBA-PD (start)
20240722_182310Pipeline of GD & GBA-PD > GBA-PD > Correction studiesAnimal models of GBA-PD tail, Correction studies (Rocha 2015 / AAV-A53T / Rockenstein 2016), S15 model, Sardi 2018 start; Animal models of GBA-PD (continued, A53T α-syn row); Correction studies; S15 model comparison; Sardi et al. 2018, PMID ?; …
20240722_182313Pipeline of GD & GBA-PD > GBA-PD > Correction studiesCorrection studies tail, Message 모음 (GBA activity / GlcCer / GlcSph / a-syn), Goals, w reduced GBA activity Gene Therapy; Correction studies (continued); Message 모음 (GBA activity-GlcCer GlcSph - a-syn); SNCA/SNCA; CBE mice / PR001; …
20240722_182316Pipeline of GD & GBA-PD > GBA-PD > GBA vs UPDRS vs MOCAPatient Selection, nGD biomarker grid, Models / Omics, BMx Milestones, Patient recruitment, GBA vs UPDRS vs MOCA; Patient Selection; nGD biomarker / study-design grid; Model; Omics / Imaging / Neurophysiologic / CSF proteomics; …
20240722_182320Pipeline of GD & GBA-PD > GBA-PD > GBA vs UPDRS vs MOCAGBA vs UPDRS vs MOCA (full literature comparison); Top longitudinal block (continued); Uncertain Spans; Clinical dev Scenarios; genotype / Activity / Activity-DBS rows; …
20240722_182323Pipeline of GD & GBA-PD > GBA-PD > GBA vs UPDRS vs MOCACognition / Motor+cog / NfL tail, Progression summary, MRI GBA, Leocadi 2022, Thaler 2018, Agosta 2013; Progression summary; MRI GBA; longitudinal PPMI (Caminiti, 2022 #2706); MRI; …
20240722_182326Pipeline of GD & GBA-PD > GBA-PD > PET Imaging GBAMRI GBA tail (Caminiti DATSCAN / Leocadi / Thaler / Agosta / Filippi), PET Imaging GBA progress; MRI GBA (continued); DATSCAN longitudinal text; Leocadi 2022 #2708 (Italy); Cortical thickness and Sub-cortical Volume; …
20240722_182330Pipeline of GD & GBA-PD > GBA-PD > PET Imaging GBAPET Imaging GBA history, Handover to Otake, Catalytic vs Allosteric binders, PET SC, GBA GT reporter table; Cf) Functional assay; History (Takeshi Wakabayashi / Otake / Makoto Fushimi); Handover to Otake-san on GBA PET; 1. History; …
20240722_182333Pipeline of GD & GBA-PD > GBA-PD > PET Imaging GBA > GBA ActivatorAADC PET tail, Rationale of GBA PET, GBA expression evaluation, DDU/MJFF PET Imaging GBA, GBA Activator PRC1; AADC PET tail; Rationale of GBA PET; GBA expression evaluation by imaging; DDU’s PET Imaging GBA; …
20240722_182336Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > RD-DDU GBA ActivatorGBA Activator criteria, Target profiles, Definition, GBA activity / GlcSph, RD-DDU GBA Activator, L444P Lyso-GL1, GBA GT start; GBA Activator criteria (continued); 20200428 Ceri meeting — Target profiles to meet the milestones (nGD); Definition (Below 20200529 LGE sDIB); GBA activity and GlcSph reduction; …
20240722_182339Pipeline of GD & GBA-PD > GBA-PD > GBA GT > Executive summaryGBA GT Executive summary cassette selection, 3m-mice ICV screen, D409V 7-group selection, TE / PRC1 / Bettina, PD mouse model 4L/PS; STIG1 — cassette selection (criterion: GBA transgene expression ≥ 1× endogenous GBA); 3m mice ICV experiment (3 groups); 7-group D409V homozygous cassette-selection study (rAAV9, ICV); MOI definition; …
20240722_182343Pipeline of GD & GBA-PD > GBA-PD > GBA GT > NHP studies > SNBR (Shin Nippon Biomedical Laboratories)In vitro GBA-PD POC, GBA-GT PK-PD strategy, Immunogenicity assays, NHP studies / SNBR; In vitro GBA-PD model (continued); GBA-GT [PK-PD strategy]; GBA GT [Immunogenicity assays] prc1; NHP studies; …
20240722_182346Pipeline of GD & GBA-PD > GBA-PD > GBA GT > TM GT GBA-PDSNBR/NHP tail, Otake-san’s update, RD-DDU GBA GT, TM GT GBA-PD planning matrix, GBA PET / mRNA / pathway; SNBR / NHP results (continued); Otake-san’s update; RD-DDU GBA GT; TM GT GBA-PD; …
20240722_182351Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > NHP Translational ModelSample size estimation, Tx Paradigm, Biomarker Usage Map, DMPK PK/PD, Translational questions, Tempo code 43/6-100; Top header carry-over; Experimental Medicine; Tx Paradigm; Biomarker Usage Map: GBA activator in PD (Takeda); …
20240722_182355Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > CompetitorGCB-BTV Executive summary tempo code, Biodistribution Studies, Comp Bio E-selectin, Competitor (Roche / BIAL LTI-291 / NCT05819359 ACTIVATE); GCB BTV Executive summary: tempo code 43/6-100; Biodistribution Studies; Comp Bio; Competitor; …
20240722_182358Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > Competitor > Fusion ProteinBIAL LTI-291-004 tail, GBA activation table, Questions to BIAL, Gain Therapeutics GT-02287, Fusion Protein anti-TfR1 sdAbs; BIAL LTI-291-004; GBA activation table; Questions to BIAL; Gain Therapeutics GT-02287; …
20240722_182402Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > RD DDU’s PET Imaging GBARD DDU’s PET Imaging GBA: GCB PET vs Lysosomotropic PET, BD study, Pharmacological modulation, Sample size estimation; PE1; GCB PET vs Lysosomotropic PET; Feasibility / BD study / Pharmacological modulation study; Sharefoldr / team meeting / 001 PE preparation; …
20240722_182406Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > Acid ceramidase inhibitorIVC / TM / GBA2, Acid ceramidase inhibitor Yuta exec summary, Acid ceramidase / Farber lipogranulomatosis genetics; GBA2; Model system; Acid Ceramidase inhibitor — Executive summary (Yuta) (PFR-4153-100); Questions / Research hypothesis; …
20240722_182409Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > GALC (galactosylceramidase) > Genetics GALC for PDFarber lipogranulomatosis (continued), IPDGC ASAH1 LSD variants table, CADD C-Score note, GALC, Genetics GALC for PD; Farber lipogranulomatosis (continued); IPDGC LSD-Gene-Variants Supplementary Table 3 (ASAH1); Cf) CADD C-Score; GALC (galactosylceramidase) (=galactocerebrosidase) (=Galcase); …
20240722_182412Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > GalactosylSph (=psychosine) > Assay of GalactosylSphGALC Genetics tail (NIH ClinVar tally), GalactosylSph (=psychosine, GalSph), Assay of GalactosylSph, Krabbe’s Disease start; NIH ClinVar Variation type / Clinical significance (GALC tail); Galactosylsph (=psychosine, GalSph); Disease state comparison; aSyn / Krabbe relationship; …
20240722_182416Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > Pipeline of KD > Target validation and correctionKrabbe psychosine continued, KD subtypes, Pipeline of KD, MOA for PD, Monthly Report (AC Inhibitor), Safety / Target validation; Krabbe psychosine continued; Pipeline of KD; MOA for PD; Monthly Report (AC Inhibitor) 202101; …
20240722_182419Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > Target validation and correction > 총정리중 GBA+PD에서 LysSafety BM tail, Kim 2018 carmofur GBA-KO results, Figure 7 / Figure 8, GalSph / psychosine summary, Krabbe questions, GALC enzyme summary; Safety BM tail; Target validation and correction — Kim 2018 #1185 carmofur GBA-KO; GalSph (psychosine) summary; Krabbe / open questions; …
20240722_182422Pipeline of GD & GBA-PD > Supplement > CNS & PNS > Cerebral CortexAIM-PD-AD Emory study, Anatomy CNS & PNS, Classification of nervous system, Spinal nerve, Cerebral Cortex layers; AIM-PD-AD (Emory University); Preliminary Results [from 제목있는 문서]; [Plan]; Anatomy — CNS & PNS; …
20240722_182426Pipeline of GD & GBA-PD > Supplement > Cerebral Cortex > Neuron > SynapsePNS / Spinal nerve / Cerebral Cortex (continued), Neuron / Synapse / Dendritic spines / PD-related changes; PNS / Somatic / Autonomic; Classification of nervous system; Spinal nerve; Cerebral Cortex (6 layers); …
20240722_182429Pipeline of GD & GBA-PD > Supplement > Neuron > Synapse > Postsynaptic density > Cranial nerveNeuron / Synapse continued, Asymmetric synapse density table, turnover, Synapse Asymmetric vs Symmetric, Postsynaptic density, Cranial nerve; Neuron; Common types of dendritic spines; Asymmetric synapse density; turnover; …
20240722_182433Pipeline of GD & GBA-PD > Supplement > Cranial nerve > Facial n > Nigrostriatal pathway > Basal GangliaCranial nerves nuclei tail, Facial n decomposition, Nigrostriatal pathway, Wakabayashi 2006 postmortem, SNc / DA neurons, Functional subdivisions of striatum, Basal Ganglia start; Cranial nerves nuclei tail; Facial n; Nigrostriatal pathway; Wakabayashi 2006 postmortem (paranigral nucleus PN, medial group MG, dorsal group DG, ventrolateral part VL); …
20240722_182436Pipeline of GD & GBA-PD > Supplement > Basal Ganglia > MBN (nucleus basalis of M…) > Development of brain (bra…) > BrainstemBasal Ganglia tail, Substantia nigra MR sequences, MBN (nucleus basalis of Meynert), Development of brain, Brainstem (Pons / LDT / Locus coeruleus / Medulla), Borghammer 2021 BODY-FIRST vs BRAIN-FIRST PD; Substantia nigra MR sequences; MBN (nucleus basalis of Meynert); Development of brain (brain vesicles); Brainstem; …
20240722_182440Pipeline of GD & GBA-PD > Supplement > Gut > Commissures > Anatomy-Neurological signs > General(Borghammer, 2021 #1484) BODY-FIRST vs BRAIN-FIRST PD, Commissures, Anatomy-Neurological signs / General, motor anatomy table, Reported signs and symptoms; (Borghammer, 2021 #1484); Panel A — Connection diagram; Panel B — Brain first; Panel C — Body first; …
20240722_182443Pipeline of GD & GBA-PD > Supplement > PD > Animal model > CROs > Database > Antibody > Epitope mappingPD neurological signs (motor + non-motor), Animal model / CROs / Database, Antibody monoclonal vs polyclonal, Epitope mapping, Affinity Maturation, APOE start; PD — Neurological signs; Animal model; CROs; Database; …
20240722_182447Pipeline of GD & GBA-PD > Supplement > Antibody > Epitope mapping > APOE > AQP4 potentiatorAQP4 potentiator (mislocalization / amyloid / p-tau), Glymphatic system in PD, AQP4 SNP, Burfeind 2017 cohort, Target validation, Ariba procurement, Astrocyte start; AQP4 potentiator; Increased amyloid accumulation in AQP4 KO × APP-PS1 mice (Mol Neurodegener. 2015, 10:58); Exacerbation of p-tau accumulation and neuronal death in AQP4 KO × PS19 mice; Glymphatic system in PD; …
20240722_182451Pipeline of GD & GBA-PD > Supplement > PET for astrocyte > aSyn > Animal models of aSyn > Non-Spreading ModelsReactive astrocytes A1/A2, (Li, 2019 #2281) molecular-expression table, PD and astrocyte, PET for astrocyte [11C]BU99008, aSyn animal models Non-Spreading + PD mouse models in QPS Line 61; Reactive astrocytes (small summary); Reactive astrocytes — Below (Li, 2019 #2281); PD and astrocyte; PET for astrocyte; …
20240722_182454Pipeline of GD & GBA-PD > Supplement > aSyn > Animal models of aSyn > Non-Spreading Models > Spreading ModelsNon-Spreading Models continued (WT Wade-Martins / F28tg / mjff / Mutant M83 PrP / KI rat / Truncated), Spreading Models, in vivo evidence, Atuka NHP results matrix; Animal models of aSyn — Non-Spreading Models (continued); Spreading Models; Methods; in vivo evidence; …
20240722_182457Pipeline of GD & GBA-PD > Supplement > aSyn > Animal models of aSyn > Spreading ModelsAtuka NHP Results matrix tail, Woodruff lab a-syn of mouse, PFF model (mouse pre-formed α-synuclein fibril), TAKEDA w Atuka Lentivirus, Gordon 2018 #587 / Chu 2019 / Luk 2012 / Duffy 2018 / Patterson 2019; Atuka NHP Results matrix (continued); Woodruff lab; PFF model (mouse, pre-formed α-synuclein fibril); Spreading models — TAKEDA w Atuka and key references
20240722_182501Pipeline of GD & GBA-PD > Supplement > aSyn > Animal models of aSyn > Non-Spreading Models > Spreading ModelsPatterson 2019 / SNCA BAC tg mouse / Bru 2013 #1370 row tail, spreading-models comparison table (PFF vs AAV with mechanism / Tx aims / Chung 2019 paragraph), Rey 2019 / 2018 / 2016 olfactory-bulb PFF rows, PFF preparation start; Spreading-models row continuation (PFF + AAV characterization); Olfactory-bulb PFF rows; PFF preparation
20240722_182504Pipeline of GD & GBA-PD > Supplement > Non-Spreading Models > Spreading Models > Summary of a-syn animal models > MOA of aSyn spreadingPFF preparation tail (storage / sonication / pilot validation), Vargas 2019 Summary of a-syn animal models Table 1, Transgenic vs Vector vs Injection matrix, MOA of aSyn spreading axon transport table, Antibodies to aSyn start; PFF preparation (continued); Summary of a-syn animal models (J.Y. Vargas et al., Ageing Research Reviews 50 (2019) 89-101); Table 1 — Summary of in vivo studies representing the major milestones in the understanding of α-syn prion-like spreading properties.; Transgenic vs Vector-mediated α-syn vs Injection comparison; …
20240722_182507Pipeline of GD & GBA-PD > Supplement > Summary of a-syn animal models > MOA of aSyn spreading > Antibodies to aSyn > Astrocytic aSynMOA of aSyn spreading tail, Antibodies to aSyn (matrix + per-antibody host/Species/epitope), Vaikath 2019 #1599 antibody fragment map, Astrocytic aSyn (Aflaki 2020 GD1/GD2 iAstrocytes), CSF & Blood Total aSyn level start; Antibodies to aSyn; Per-antibody host / Species / epitope; Astrocytic aSyn; CSF & Blood (a-syn)
20240722_182510Pipeline of GD & GBA-PD > Supplement > MOA of aSyn spreading > Antibodies to aSyn > Astrocytic aSyn > CSF & Blood (a-syn)CSF & Blood (a-syn) — total aSyn levels, Why reduced in CSF? paragraph, King 2022 / Eusebi 2017 / Chou 2014 / Mollenhauer 2017 / Hall 2016 / Hansson 2014 / Compta 2015 / Majbour 2021 DenoPa / van Steenoven 2018 VUMC studies; Why reduced in CSF?; Total aSyn level — study comparison; Uncertain Spans
20240722_182514Pipeline of GD & GBA-PD > Supplement > Astrocytic aSyn > CSF & Blood (a-syn) > Distribution > Expression of aSynCSF & Blood (a-syn) study rows continued (Schirinzi 2019 / Wang 2012 / PD01A / Lin 2019 / Chang 2019 / Wang 2020 / Chatterjee 2020 / Yamashita 2023 / Transpharmation Attyloid Blömeke 2022), Js summary table, MDS 2020 PD01A note, Distribution, Expression of aSyn, Bulk tissue SNCA violin plot, Postmortem brain study rows; CSF & Blood (a-syn) — study rows continued; Summary; Distribution; Expression of aSyn; …
20240722_182518Pipeline of GD & GBA-PD > Supplement > Function (of normal aSyn) > Grading/Scoring of LB pathology > Homology > In vitro studies of aSynFunction (of normal aSyn), Grading/Scoring of LB pathology, Homology, In vitro studies of aSyn matrix, In vitro pharmacology assays; Function (of normal aSyn); Grading/Scoring of LB pathology; Homology; In vitro studies of aSyn; …
20240722_182522Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aS… > Oligodendrocytic aSyn > PET aSynLewy Body / components / Two types / Toxic mechanisms / Lewy neurites, Lipid-aSyn interaction review, Mutations SNCA, Neurodegeneration and aSyn (Mori 2006 + Lee 2017 staging table), DBM, Oligodendrocytic aSyn, PET aSyn vendor matrix start; Lewy Body; Two types of LBs; Toxic mechanisms; Lewy neurites; …
20240722_182524Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aSyn > Oligodendrocytic aSyn > PET aSynPET aSyn vendor matrix (AC Immune / MODAG / University of Zurich / QST / Shenzhen / Merck / Mass Generic at Brigham) — Lead compound, Derivative, Structure, patient, Timeline, detects, in vitro Saturation binding affinity, IF, ARG, Preclinical Tox start; PET aSyn vendor matrix; Preclinical Tox (start)
20240722_182528Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aSyn > Oligodendrocytic aSyn > PET aSynPET aSyn vendor matrix tail (Animal Cold/Hot, Animal in vivo PET, ARG, Preclinical Tox 2-rat studies, FH Protocol Eval, Protocol B-safety, FH F/U + team note, Four αSyn isomers, Alzforum half-life note); PET aSyn vendor matrix (continued); Alzforum potential booster
20240722_182532Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aSyn > Oligodendrocytic aSyn > PET aSynPET aSyn vendor matrix (Takeda CS 20230427, AlzForum FOSO28, MJF supported, Method comparison Smith 2023, SPAL-T-06 vs C05-05, Round 2 compound), Takeda co-owner license agreements; PET aSyn — vendor matrix continued; SPAL-T-06 vs C05-05; Takeda is co-owner of this asset; co-owner block
20240722_182535Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aSyn > Oligodendrocytic aSyn > PET aSynPET aSyn — QST Specified Clinical Trial planning (Operational lead, Funding MJF, Schedule, IND, Goal, Protocol, Process, Contract, Freedom to operate, GLP-Tox); QST Specified Clinical Trial — planning (PET aSyn vendor matrix continuation)
20240722_182539Pipeline of GD & GBA-PD > Supplement > Neurodegeneration and aSyn > Oligodendrocytic aSyn > PET aSyn > Postmortem aSynQST PET aSyn — process / contract / freedom to operate / GLP-Tox 20230808 plan, 20230714 Biomarker FlytoAs, Postmortem aSyn opener; GLP-Tox; (20230714) 3. Biomarker FlytoAs; Postmortem aSyn; Uncertain Spans
20240722_182542Pipeline of GD & GBA-PD > Supplement > Postmortem aSyn > Release > Safety for aSyn > KO of α-syn > Seed-Amplification AssayPostmortem aSyn detailed staging table tail, Release of α-syn (sulfonylurea/SUR1 channel), Safety for aSyn / KO of α-syn (SNCA KO mice + Vilar 2018 + Markovich 2007), Seed-Amplification Assay (RT-QuIC) Materials/96 well/Real-time readout/Limitation/Advantage, vendor matrix (PPMI/Tichi/Penix/OPDC/Lema-Marambio/Caughey); Postmortem aSyn — staging table (continued); Release; Safety for aSyn; KO of α-syn; …
20240722_182545Pipeline of GD & GBA-PD > Supplement > Safety for aSyn > KO of α-syn > Seed-Amplification Assay > QuantificationSeed-Amplification Assay (RT-QuIC) — vendor / study rows (Russo 2020 / Onia 2021 / Pankha 2014 / Arnold 2021 / Brown 2023 / Brookmeyer 2021 / Olucium 2023 / Groves 2018), Quantification opener (Kinetics readouts, Single-seed level, PrP-QuIC modeling, factors influencing lag time); Seed-Amplification Assay (RT-QuIC) — study rows; Quantification; Kinetics readouts; method (Quantification)
20240722_182549Pipeline of GD & GBA-PD > Supplement > Seed-Amplification Assay > Quantification > Utility > Sources of aSynQuantification continued, end-point dilution + Calibration curve, application, Utility (Sidoroy 2023 Syn-target / RRK2-targeted / Aa-targeted), MJF workshop 20220421, Sources of aSyn (Cell/Brain/Recombinant/Synthetic + Aβ Figure 1); Quantification (continued); Utility; MJF workshop 20220421; Sources of aSyn
20240722_182552Pipeline of GD & GBA-PD > Supplement > Sources of aSyn > Skin aSyn > Species > Native conformation > Summary aSyn speciesSources of aSyn detailed table (Recombinant / cell culture / Brain extract / Chemical synthetic with Method / pros / Product / parameter modelling rows), Skin aSyn (Syn-One test, Vasiliev 2018, Donadio 2024 #2752), Species / Native conformation, Primary aSyn species table (monomeric / tetramer / pSer129 / Misfolded / β-pleated sheet / Insoluble / Oligomeric / protofibril / amyloid-like fibril / Lewy bodies); Sources of aSyn — detail table; Skin aSyn; Species; a-syn primary conformation; …
20240722_182556Pipeline of GD & GBA-PD > Supplement > Summary aSyn species > NDE > Fragments (Truncation) > Assay TruncationPrimary aSyn species table tail, BDD update 202305 N-terminal α-synuclein, NDE (TAK-341 IDP / ADx + Cooveohe collaborations), Analyt-NIA ELISA SG4, Fragments / Splice variation (C-terminal truncation 10-30%), Assay Truncation (LC-MS isoform characterization); Primary aSyn species — table tail (continued); (BDD monthly update 202305) — BDD - N-terminal α-synuclein (Cterminal); NDE (Neuronal-derived exosome); Analyt-NIA ELISA (SG4); …
20240722_182559Pipeline of GD & GBA-PD > Supplement > Summary aSyn species > NDE > Fragments (Truncation) > Assay TruncationAssay Truncation continued (BDD update 202404, Kahn 2024 #2973 LC/MS isoforms in cingulate cortex), SNCA truncation isoforms (-126/-112/-98 with theoretical MW/aa), Bluhm 2021 #2157 schematic protease cleavage map (trypsin/MMP-3/Calpain-1/Cathepsin D/Neurosin/casp1), Sorrentino 2020 schematic, binding site 102-130 abundance table; Assay Truncation (continued); SNCA truncation isoforms; (Bluhm, 2021 #2157); (4.1 binding site 102-130) — abundance table
20240722_182602Pipeline of GD & GBA-PD > Supplement > NDE > Fragments (Truncation) > Assay Truncation > Structure of aSynHalia 2014 #2073 truncation table tail, Splice variation (4 αSyn transcripts SNCA-140/-126/-112/-98), Beyer 2008 Neurogenetics alignment, TAK-341 epitope on exon 5, Pathological Conversion + Pathological Aggregation cycle figure, Structure of aSyn opener, Brain Res 2012 1432:90 α-synuclein domain figure, 4q21.3-q22 chromosome diagram; (Halia, 2014 #2073) — truncation isoform table tail; Splice variation; Pathological Conversion → Pathological Aggregation cycle; Structure of aSyn; …
20240722_182606Pipeline of GD & GBA-PD > Supplement > Assay Truncation > Structure of aSyn > Synapse and aSyn > TM for aSyn programsThree major αsyn domains (N-terminal / NAC / C-terminal acidic tail), Synapse and aSyn (Calleri 2015 90% presynaptic deposits), TM for aSyn programs (Monomer / Pansson 2018 fibril CDMS, αSyn oligomer enrichment plan, paSyn / Fragments / LB / PD vs MSA / DLB SPAL? rows); Three major αsyn domains; Synapse and aSyn; TM (target modulation) for aSyn programs; PD vs MSA / DLB ? (SPAL?)
20240722_182609Pipeline of GD & GBA-PD > Supplement > Synapse and aSyn > TM for aSyn programs > Turnover of aSyn > ATP13A2 (Park9)TM for aSyn programs continued (TAK-341 in hippo / SNCA MEDI-1341 / SNCA p3 / How long?), Turnover of αSyn (Stefano 2018 #770 review with αSyn 240h half-life table by macroautophagy / cathepsin D / lysosomal pathways), ATP13A2 (Park9) opener (gene/structure, KRD, NCL, Heterozygous carriers, ATP13A2 in Lewy bodies); TM for aSyn programs (continued); Turnover of αSyn; ATP13A2 (Park9); NCL; …
20240722_182613Pipeline of GD & GBA-PD > Supplement > ATP13A2 (Park9) > BBB / disruption / opener > BD > Process / BeyondBio > BiobankATP13A2 (Park9) MUA + Animal model + ATSURO MICRO + PXEAEAU Lysosomal protein degradation + SAL BAC + MPO score + Disruption of BBB + BBB disruption in PD + BBB opener Cordance + non-confidential CDA + BD/Process/BeyondBio + Biobank + PPMI; ATP13A2 (Park9) — MUA; Animal model; ATSURO MICRO; BUCITAL DIO; …
20240722_182617Pipeline of GD & GBA-PD > Supplement > Process > BeyondBio > Biobank > MJFPPMI Subjects/Available Data/Biospecimens, Shahn Rahmadhan + Por 20240228 Samantha notes, BioFiND, LRRK2 Cohort Consortium (LCC) Cross-sectional + Longitudinal + 23&Me Studies, S4 Systemic Syn Sampling Study (Chahine 2020 #1605), Cf SC12 antibody note, Nilo-PD opener; PPMI; BioFiND; Available Biospecimens and Data; LRRK2 Cohort Consortium (LCC); …
20240722_182620Pipeline of GD & GBA-PD > Supplement > BeyondBio > Biobank > MJF > Netherlands Brain BankNilo-PD continued (Cohort 1/2 + Available Data + Biospecimens), MJFF PPMI iPSC LINES, MJFF biobank contacts, 14 Types of Samples Available, Cohort Consortium ETBNRPP, tranSMART panel, Netherlands Brain Bank opener (autopsy procedures + paraffin embedded samples); Nilo-PD (continued); MJFF PPMI iPSC LINES; MJFF biobank — contacts; 14 Types of Samples Available; …
20240722_182624Pipeline of GD & GBA-PD > Supplement > BeyondBio > Biobank > MJF > Netherlands Brain BankNetherlands Brain Bank tissue request continued (Sample transfer process / MTA / Transportation / cost per sample / IHC paraffin vs frozen / Molecular analysis / Location-Region-Analysis-Brain Amount table / Tony Wolinsky purchasing flow / NBB blood-cells correction / genetic testing / segregation in the family / pathogen testing / NLRP3 plan / AD co-pathologies / Braak stage classification opener); Netherlands Brain Bank — tissue request flow; Transportation; cost per sample; IHC paraffin preferred / frozen + Tissue; …
20240722_182627Pipeline of GD & GBA-PD > Supplement > Biobank > MJF > Netherlands Brain Bank > Imperial College Brain BankBraak staging diagram tail (Thal 2002 + CERAD), Imperial College Brain Bank (Parkinson’s UK contacts + sample request + section types + tissue collections + materials + Jaewon QA), ~400 genotyped PD cases table opener; Braak staging diagram (continued); Imperial College Brain Bank; Controls definition; Section types; …
20240722_182630Pipeline of GD & GBA-PD > Supplement > MJF > Netherlands Brain Bank > Imperial College Brain Bank > Other BiobanksImperial College Brain Bank PD genotyping table tail, sample availability + USDA statement, Imperial College Brain Bank Ariba PO process, Other Biobanks (NCNP J-PPMI Niigata univ TMM Cohort Study); Imperial College Brain Bank — sample availability; Other Biobanks; J-PPMI (Juntendo Univ); Niigata univ — Postmortem brain PD aSyn fibril; …
20240722_182634Pipeline of GD & GBA-PD > Supplement > MJF > Netherlands Brain Bank > Imperial College Brain Bank > Other BiobanksUK Brain Bank Network (NBB), BDR / NeuroChip / Camp dataset access table, NIH NeuroBiBank, NCRAD + EUFortuv MICAgou, PrecisionMed, BIOIVT hemoglobin, fibroblast vendors (NINOS / Cell Applications / Lonza / Lifeline / promogen / Sanjusbo / LEDBO / Cellbio / pmgenel), Biochain catalog, BioSEND, Novus Biologicals NB820-59177; UK Brain Bank Network (NBB); BDR access table; NIH NeuroBiBank; NCRAD (National Centralized Repository for Alzheimer’s Disease and Related Dementias); …
20240722_182637Pipeline of GD & GBA-PD > Supplement > Netherlands Brain Bank > Imperial College Brain Bank > Other Biobanks > Biobanks in JapanOther Biobanks register continued (BIOIVT, vendors, Biochain, BioSEND, Novus Biologicals, ACCEGEN, Genomics England Banner Brain, NJ Brain Endowment Bank, NDRI, Neurobiobank Munich), Korean Open Government License KOGL Type 4, J-PPMI NCNP, Biobanks in Japan table; Other Biobanks register (continued); Novus Biologicals — NB820-59177 (continued); ACCEGEN; Genomics England Banner Brain; …
20240722_182641Pipeline of GD & GBA-PD > Supplement > Imperial College Brain Bank > Other Biobanks > Biobanks in Japan > BIOMARKERBiobanks in Japan tail (Tsukuba1 + Kyoto-U Nagahama phase0), 20231009 Hooki Tsmurni Niigata univ, BWMVRINER NSTM BM Tracking (KPI) matrix, GBA Activity / GBM / GACer assay rows, CSF Sample (Sato) prototype, CSF & NDE row, ARM lysosomal marker discussion; Biobanks in Japan (continued); 20231009 Hooki Tsmurni Niigata univ; BWMVRINER NSTM BM Tracking (KPI); CSF Sample (Sato); …
20240722_182644Pipeline of GD & GBA-PD > Supplement > Imperial College Brain Bank > Other Biobanks > Biobanks in Japan > BIOMARKERBIOMARKER tracker matrix continuation — lysosomal markers + aSyn / GlcSph / SCSF assay rows, Annapurna note, C8H CSF LCMS, Dr Margreith UMCS oligo aSyn discussion, MID/ELISA Parkin assay (cGAMP / pSer65-Ub), CSF Buffer 125 pg/mL row + Migdalisa Richards Ciegg patient sample notes; BIOMARKER tracker — lysosomal markers + aSyn; GlcSph (Sato); GlcSph & GalSph (Sato) — assay status; C8H CSF LCMS; …
20240722_182704Pipeline of GD & GBA-PD > Supplement > Imperial College Brain Bank > Other Biobanks > Biobanks in Japan > BIOMARKERBIOMARKER tracker continued — pSer65-Ub MJ assay (Atalee + SMC4Ps + Boston Bioscience LSBio antibody), MITOKININ NBB CSF, BoMM 240 NBB samples, NLM newsletter PhanoSwitch saliva-EV proteomics, Korean RDDDU/OCT decision note; BIOMARKER tracker (continued); pSer65-Ub MJ assay updates; pSer65-Ub assay sensitivity / quantitation; MITOKININ NBB CSF; …
20240722_182708Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestraBIOMARKER tracker continued (Oxygen consumption + LC3 II), Stage / Date / Deliverables panel, BMx Platform / BPS / Feasibility / BFA / Technical / BTV / IVC / GPT / Tox / BFP stage matrix, BioOrchestra opener (B amyloid CD36 microglia, miR-485-3p, ELAV12 / Sckeckel 2016 #1591); BIOMARKER tracker (continued); Stage / Date / Deliverables (small panel); Wide tracker matrix; BioOrchestra (start)
20240722_182711Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > BloodBioOrchestra ELAVL2 / miR-485-3p target table (continued), nELAVL bullet notes, Blood section opener (WBC / PBMC / plasma table, innate vs adaptive immunity, blood sample handling); ELAVL2 / miR-485-3p target table (continued); Blood; Uncertain Spans
20240722_182715Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > BloodBlood sample table tail (Suitable for RT PCR? / transcriptomics), Genomics England sample-handling guidance, Plasma / Serum / PBMC collection workflows, Lifespan / structure opener (classical monocytes emerge first from marrow); Blood sample table (continued); Plasma; Serum; PBMC (and PLASMA); …
20240722_182718Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Blood > PBMC > Vendors of PBMCCell EM diagram citation, Lysosome / Example / 없는 것 table, mitochondria & aSyn notes, Sample preparation and process, Thawing and resting, Culture of PBMC, Vendors of PBMC table opening; Vendors of PBMC; Uncertain Spans
20240722_182722Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Braak StagingVendors prospective tail, Braak Staging table (1-6 / HindBrain → Neocortex), Dx prodromal narrative, Zheng 2019 arrival-time chart, Masuda-Suzukake α-syn fibrils diagram, Takeda Braak / Propagation slides; Braak Staging; Abnormal α-syn fibrils (intracerebral injection to SN); Braak Staging of Parkinson’s Disease (Takeda slide); Propagation of α-synuclein Pathology: Human (PD) (Takeda slide); …
20240722_182725Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Braak StagingBurke 2008 H&Y vs Braak Stage scatter plot, Calcium channels (Cav subtypes table), SN DA neurons / Isradipine / In PD notes, askIT / CDA email thread (start); Fig. 3; Fig. 2; Calcium; CDA / askIT email thread (start); …
20240722_182728Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI > Companies CEICDA email thread tail, CEI / Companies CEI / Vesalius distinct clinical feature types, Ambagon Argo molecular-glue 14-3-3 client table, Brain / CSF detection table, Giusto 2021 14-3-3 review, Underwood 2021 PFF mice notes; CDA email thread (continued); CEI; Companies CEI; Uncertain Spans
20240722_182732Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI > Companies CEIUnderwood 2713 PFF tail, Zhao/Dzamko/muda LRRK2 14-3-3 notes, Ambagon-Takeda aSyn R&Rs confidential, 14-3-3 isoform/YWHA table, Deal concept / R&R, Mayor (AC immune) program, Competitor list, aSyn PET, Jaewon’s Lucy Tx Q&A, Samrara Tx Triage, Individual report (T3D / Neuracle / Nuravax); Mayor = AC immune; Samrara; Individual report (start); Uncertain Spans
20240722_182735Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI > Companies CEICompanies CEI / Individual report (T3D, Neuracle, Nuravax, Escape Bio, Amsterdam Neuroscience, Muna Tx, GNS Healthcare, BlueRock, Genuv, Lysogene, Sunnybrook, Vincent / TMEM175, Annovis Bio, amimod, ADRx); Individual report; Uncertain Spans
20240722_182738Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI > Companies CEICompanies CEI / Individual report (continued): amimod hepatotox tail, ADRx tau, Nuravax follow-up, NeuroPn, Yumanity, Athira, Innomedica, Kyowa Kirin, BioVie NE3107, Logos Bio ADAD gene therapy / OGA, Creus CxCR7, Neuroinitiative Parkin activator, Tigen Minipig RD, Neurolixis NLX-112 / NLX-101 / NLX-204, Tx Nurr1:RXRa program (BRF110); Individual report (continued); Tx — Nurr1:RXRa program (BRF110); Uncertain Spans
20240722_182742Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI > Summary CEITx Nurr1:RXRa BRF110 program tail (TE / Neuroprotection / aSyn / neuroinflammation rows), Seelos / Herophilus / Myro Tx / AcureX / VesperBio / Priavoid / D&D Pharma (Neuraly), GLP-1R agonists clinical trials table, Summary CEI section opener; Tx — Nurr1:RXRa program (BRF110); Companies CEI; Summary CEI; Projects in DD/Partnering Discussions:; …
20240722_182745Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI > Summary CEISummary CEI continued: Confidential stage opportunities, Opportunities recently sent for triage, Relook at past MONITOR/Decline, NeuroD Landscape Refresh, NeuroD Heat Map, 20220906 Triage live discussion (Cerevel / Neuron23 / Creus / Ionis / NLRP3 group / Transitions Bio / Wren / Fluidic / AC Immune / Blue Rock), Key Projects Rundown, Heat Map / Competitive Landscape, SE Core Team Meeting agenda; Confidential stage opportunities and upcoming evaluation meetings; Opportunities recently sent for triage:; Relook at past MONITOR/Decline triage decisions:; NeuroD Landscape Refresh:; …
20240722_182749Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI > Summary CEISummary CEI continued: Key Projects Rundown / Heat Map / SE Core Team agenda recap, NeuroD SE Core Team Meeting (November 1, 2022) topics, JP Morgan Planning, Project Torchlight / Apex / Turing / Bon / Link rundown, Late Stage Scan / Prescouter, 21130 protein-degrader landscape (C4 / Plexium / Kymera / Neomorph / Wren / Anima Biotech), Meetings CEI / heat-map CEI sharepoint links; Key Projects Rundown; Heat Map / Competitive Landscape (recap); NeuroD SE Core Team Meeting; Topics; …
20240722_182752Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI > Cerevance > DeliverablesSummary CEI tail (Anima Biotech / Hi Julia), Heatmap CEI / Landscape / Meetings CEI sharepoint links, Cell lines (Neuronal catecholaminergic / Non-catecholaminergic / Non-neuronal tables), Cerevance Datasite & Deliverables (Project Acorn / CVN424 GPR6 inverse agonist) review table; Heatmap CEI; Landscape; Meetings CEI; Cell lines; …
20240722_182755Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI > Cerevance > TeamCerevance Pipeline (CVN424 / CVN766 / CVN293 / CD38 / KCNJ6 / GPR65), Current Portfolio Status: April 2022 timeline, Key worlds (evaluation / scientific confidence / Opportunity), Team table (function ↔ team member); Pipeline; Current Portfolio Status: April 2022; Key worlds; Team; …
20240722_182758Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Circuits > Direct pathwayCerevance Team table (full), Centrifuge / ultracentrifuge note (Olga), Ceri Davies / Christine Klein meeting block, Mitochondrial biomarkers mini-table, Circuits / Direct pathway (cortex → striatum → SNc / GPe / GPi/SNr / STN → thalamus diagrams); Team; Centrifuge; Ceri Davies; Christine Klein; …
20240722_182802Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Circuits > Indirect pathwayHealthy / Parkinson’s disease comparison panels, Direct pathway PD data table (SNc / D1 MSNs / GPi&SNR / thalamus / Cortex), Indirect pathway data table (D2 MSNs / GPe / STN / GPi&SNR / thalamus), DBS / GAD note, Input / Output diagrams, PD / HD cause-direct-indirect-consequence table, In HD subsection (D2 MSN / D1 MSN), Mhtt synaptic notes, Hypokinetic Sx; Healthy vs Parkinson’s disease (panel labels); Direct pathway (continued); Indirect pathway; Pathway summary; …
20240722_182805Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dose finding > Dose selection/findingHypokinetic / Hyperkinetic Sx tail, Decision-making row, Clinical trial Examples of PD (SAD), MAD, Finding (study in the target population), Concomitant Medication, Dose finding / Dose ranging study, Dose selection/finding study; Clinical trial Examples of PD; SAD; MAD; nding (study in the target population); …
20240722_182808Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dose finding > Sample size calculationC-Path SWEDD vs DAT-Deficient slide (Conrado 2018), Summary from below percentages, Description table (intervention-based suppression of disease progression), WATCH-PD sample-size narrative, 2017 Simuni Table 1, 2018 Marek Column A table, TAK-341 IDP / ePOC / GBA GT PRC1 / 20210122_RSLT_GBA GT v2_GP rows; Rate of Motor Decline in SWEDD and DAT-Deficient Subjects was Different - Statistically and Clinically (slide); Summary from below; 바람직한 description: x% (intervention-based) suppression (or reduction in) of disease progression (relative to placebo); Uncertain Spans
20240722_182811Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dose finding > Delayed start designGBA GT v2_GP sample-size tail, 20190423 Sclareol-NS imaging strategy 4-panel chart, Sample size calculation (proportion / Visions Pharmaceutical 2021 #1755 / Lundkya 2013), Innovative design / Interim analysis / β regression model / FDA-EMA principle, C-Path DAT Enrichment slide, Delayed start design (Liu-Seifert 2015 sketch), MEAN STRIATUM DAT vs AV133 table; DAT Enrichment allows ~24% Reduction of Trial Size to Detect a Disease modifying Drug Effect with 80% Power (slide); Delayed start design; MEAN STRIATUM DAT vs AV133; Uncertain Spans
20240722_182815Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > PrevalenceAV133 sample-size tail, Cognition / Cholinergic / Anatomy (basal forebrain BF corticopetal projection / PPN-LDTC / MVN / striatal cholinergic interneurons), Pathway PET enzyme table (ChAT → VAChT → AChE → SDHACU), CUMULATIVE PREVALENCE OF COGNITIVE AND PSYCHIATRIC DISORDERS OR TREATMENT IN PD (Variable / Baseline / Year 1-5); Cognition / Cholinergic; Anatomy; Pathway, PET; CUMULATIVE PREVALENCE OF COGNITIVE AND PSYCHIATRIC DISORDERS OR TREATMENT IN PD; …
20240722_182818Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > vMRICumulative prevalence summary slide (50-70% / 25% bullets), vMRI literature matrix (Burton / Ramirez-Ruiz / Xia / Melzer / Goldsby / Apostolova / Mak studies); CUMULATIVE PREVALENCE OF COGNITIVE AND PSYCHIATRIC DISORDERS OR TREATMENT IN PD; vMRI; Uncertain Spans
20240722_182821Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > PathologyvMRI tail (Segura 2018 cortical thickness), Aβ NPT brain/CSF, dual-syndrome hypothesis (fronto-striatal vs cholinergic posterior), Imaging (Aβ in PD / Forrencys 2018 / Florencys 2016 postmortem), sMRI Definition (SBM / VBM / SbM / DBM), Lewy Body score (composite / clinical / SCORE / Table FC), Pathology section (Hall 2014 postmortem); Imaging; sMRI; Lewy Body score; Pathology; …
20240722_182824Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > ConsortiumPathology tail (basal forebrain / hippocampal cholinergic system / Frontal cortex / ChAT activity), Hellinger 2008 review postmortem note, Consortium (MOBILIZE-PD / Verily / Takeda Japan / Watch / IDEA-NeXT / AMP-PD), PPMI (Enrollment / LP / Genetics Consensus / Visit / DaTscan completeness), Operations Manuals & Specimen Processing Maps, Other PPMI Program Protocols, Data & Biospecimen Use Agreements; Consortium; PPMI (Parkinson’s Progressive Markers Initiative); Study Documentation; Operations Manuals and Specimen Processing Maps; …
20240722_182827Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > ConsortiumPPMI Study Documentation / Operations Manuals overlap, Other PPMI Program Protocols, Data and Biospecimen Use Agreements and Policies, LBD (Aria 2020 #2728) sub-study, Innovative Medicines Initiative (CSF Acoustic Kit / Olink / Complement Luminex / Somalogic SomaScan 7K), EUbOPEN, PD BRAC access flow (NINDS BioSEND / Harvard Biomarkers Study / MJFF Cohorts BioFIND / DATATOP / FS-ZONE / LRRK2 / SURE-PD), MJFF Grant Portal opener (MJFF-019845 MC1 PET); Data and Biospecimen Use Agreements and Policies; Sub-Studies; LBD (Lewy Body Dementia); Innovative Medicines Initiative; …
20240722_182830Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > ContactsMJFF Grant Portal block (Pre-Proposals / Coordinating Lead PI / Full Proposal Invitations), MJF imaging meeting along with MJFF, WORKSHOP 202111 day2 conference slide, Canada / PDGENeration / Stephenson 2019 / Rare Disease Data Registry of Japan / Dr. Takeda AMED contacts, ATCs (Contacts) table opener (Soon Chung / Riley Frilghity / Rae Estrella / Won Lee / Jaewon Lee / Jaya Rosen / Jeffrey Knight / Leonis Papakosta / Zach McQuade / Toro Tomimatsu); MJFF Grant Portal — Outcome Measures; Can you switch PI’s after submitting a Pre-Proposal?; What does it mean to be a Coordinating Lead Principal Investigator?; MJF imaging meeting along with MJFF; …
20240722_182833Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > CSFRare Disease Data Registry tail / Dr. Takeda AMED tail, Contacts (extended), MTM Contact info, Cortellis Drug Discovery Intelligence (Sequence search BlastP/BlastN), CROs (오시리스 사 / KBio Health / ATECH / WA BIO), Cryofluorescence tomography (CFT) slide opener, CSF section opener (immunoassay volume notes / GICph); Contacts (continued); MTM Contact info; Cortellis; CROs; …
20240722_182836Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > CSF FlowCSF Flow / Blood contamination / Fluid compartments / Barriers in the brain; Blood contamination; CSF Flow; Fluid compartments in the brain; Barriers in the brain
20240722_182839Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > Human CSF samplingBrain CSF illustrations / Barriers table overlap, Brain structure (ECF / parenchyma / ventricular system), CSF turnover (4×/day), SE as a Surrogate for Assessing CNS Exposure (Lamotrigine / Phenytoin / Tiagabine / Acelagon AUC matrix), Sources of CSF proteins, Antemortem vs postmortem (Subhash 2008), Human CSF sampling (lumbar puncture 15-20 mL / 30 mL maximum), Cisterna magna puncture vs Catheter implantation, rhesus macaque CSF (Clingerman 2010 #1819), Serial sampling opener; Brain structure; Turnover; SE as a Surrogate for Assessing CNS Exposure: An Industrial Perspective; Sources of CSF proteins; …
20240722_182842Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > Interferons > interferon-stimulated geneMouse CSF / Rat CSF serial sampling tables, Cytokines Classification (Interleukins / Chemokines / Interferons / TNF / Colony Stimulating / TGFβ), Interleukins (IL-1β / IL-6 / IL-9 NLRP3 narrative), Interferons (Type I / Type II / IFN-γ MHC class II), interferon-stimulated gene (ISG) MOA (JAK-STAT / IFNAR / ↑ ISG gene expression), Multiplex cytokine assay (Mouse / Rat / Human Luminex / Olink panels); Serial sampling — Examples; MOUSE CSF; Rat CSF; Cytokines; …
20240722_182845Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > Multiplex cytokine assayInterferons / ISG overlap, Multiplex cytokine assay (R&D Systems Luminex 50uL / Proteome Profiler Rat XL / Olink 48 mouse panel / 96 mouse exploratory / 92 human / pricing / detectability), DBS section opener (Mid PD EARLYSTIM 2y f/u Schuepbach 2013 #1892); Multiplex cytokine assay; DBS
20240722_182848Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > Diagnosis of PDOlink panels overlap, D&D Phamatech (NLY01 NCT04154072), DBS (EARLYSTIM Schuepbach 2013 + Indication / Side effects / Prognosis bullets), DDC (Aromatic L-amino acid decarboxylase): AADC enzyme / Keaton 2023 / Perera 2023 / Petlawski 2023 / Cieselska 2017 / 18F-L-DOPA PET (Calabria 2016), Diagnosis of PD (MDS criteria / Postuma 2017 / Geils’s criteria opener); D&D Phamatech; DBS; Indication; Side effects; …
20240722_182851Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > Digital TrialsDDC AADC overlap (Cieselska 2017 / 18F-L-DOPA / Why increase / Qs), Diagnosis of PD overlap, Stages of early PD overlap, Digital section opener (Lipsmeier 2018 PMID 29701258 / 6-month Phase 1b smartphone trial / APDM composite motor endpoint), Konectom Roche-Biogen partnership, Digital Trials APDM ML feature reduction (122 → 28 → 9 → Random Forest 25 features), WATCH-PD Takeda + Biogen + MJFF program, APDM walking-feature characteristic table opener (Ratio of peak power / spontaneous tremor detection); Digital; Lipsmeier et al. 2018, PMID 29701258 / Clinical P1; goal; Validation required; …
20240722_182854Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > Digital TrialsAPDM ML / Lipsmeier 2022 / WATCH-PD overlap, APDM walking-feature characteristic (Tremor / Bradykinesia / Postural / Mobility / Speech), Timed walking / Stand and go / Postural sway / standing walk / cadence sub-tables, Issues block, MDS-UPDRS Items vs Subitems coverage matrix (Arm swing / Stance time / Cadence / Double support time / Turn duration), (Adams, 2022 #2373) Supplemental Table 2 (Wearable Sensoria / Smartphone / Smartwatch device-assessment matrix); APDM walking-feature characteristic; Issues; MDS-UPDRS Items vs Subitems coverage matrix; (Adams, 2022 #2373) Supplemental Table 2. Description and location of assessments conducted with the digital devices used in the study
20240722_182857Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > Digital TrialsAdams 2022 supplemental table tail, NCT06219629 LEARNS observational basket study, TAK071-2002 crossover trial, TAK994-2001 PK/safety phase 2 trial, TAK-341 IMED1341 MAD Apple Watch feasibility, NEA-FAST scope, Verily watch (google) JMO observational study, mObILiZe-D consortium / Bre-One uLIZE-D Apple Watch+McRoberts+Dynaport feasibility opener
20240722_182900Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > Disease area TM strategyWATCH-PD/Konektm overlap, SWO-001 Japan / CarefoOne Apple Watch ongoing rows, (Drowy, 2020 #1223) Comparison of drug pharmacotherapy survey table, Digital PD Summary (US/EU vs Japan studies), Disease area TM strategy (Mitochondria / Neurodegeneration / POC framework / Human sample / Disease modelling), Project relevance / Candidate BM / Assay development matrix (α-Syn CSF / α-Syn PET / α-Syn Retina / lysosomal markers / CSF p65-Ub / functional mitochondrial CSF BM / NAD+ 31P MRS imaging); (Drowy, 2020 #1223) Comparison of drug pharmacotherapy in Parkinson’s disease — Table 1; Digital PD / Summary; Disease area TM strategy; Uncertain Spans
20240722_182904Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > DLB vs PDD vs MSADisease area biomarker table tail (CSF p65-Ub / Functional mito CSF BM / imaging / α-syn / Lysosome rows), Omics resources (Silberring 2010 #1148), DJ-1 (Park7) block, DLB vs PDD vs MSA comparison table opener (Prevalence / Incidence / Onset / Life expectancy / Dementia / RBD / Visual hallucination / Parkinsonism / Autonomic Sx / Four core indicative diagnostic biomarkers / Supportive / Clinical Dx / DATscan / Mokette / Nocker reference table); Disease area TM strategy (continued); Omics; DJ-1 (Park7); DLB vs PDD vs MSA; …
20240722_182907Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > DLB vs PDD vs MSADLB vs PDD vs MSA imaging / pathology rows (VMAT PET / sMRI Burton-Borroni-Cohn / aSyn PET DWI Pellecchia / Synuclein-only inclusions Olivera 2021 / Aβ load Hepp 2018 / NFT / Brainstem-Limbic-neocortical / Chia 2021 GWAS / GBA mutations and MSA)
20240722_182910Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Cholinergic > DNA damageDLB vs PDD vs MSA tail (Aβ / NFT / GWAS / GBA / SNCA / CSF αsyn / Clinical trial), MSA agent registry (Lithium / Stem cell / Mesenex αSyn immunotx / Bicheron verepinstat), DNA damage section opener (definition / Causes / Type-Repair table opener / Consequence DDR / Major pathways of DNA repair table opener); DLB vs PDD vs MSA — Clinical trial implications; DNA damage; Causes; type; …
20240722_182913Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopamine system > Dopamine metabolites in PDDNA repair tail (Homology-directed / Non-homologous / Microhomology-mediated / Direct reversal / Translesion synthesis), DNA damage in PD (Sariguchi 2021 #1782 γH3AX / DNA glycosylase upregulation / NTH-1 decline with age), MOA in PD (BER capacity / SSB intermediates), Dopamine system / Catecholamine pathway (L-DOPA → DA → DOPAC / 3-MT / HVA / MAO / COMT illustrations + 18F-DOPA tracer), Dopamine metabolites in PD opener (Beriheimer 1973 #1749 postmortem 39 PD; LeWitt 2011 #1782 DATATOP CSF); DNA damage in PD; MOA in PD; Dopamine system; Catecholamine pathway; …
20240722_182916Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopamine system > Summary of dopamine systemDopamine metabolites in PD — CSF / blood / urine correlation rows (LeWitt 1992 / Stefani 2017 / Wichi 2021), Anderson 2017 DA-HVA L-DOPA narrative, Dormant dopaminergic neurons (Heo 2020 astrocytic GABA / optogenetic SNpc rescue), Summary of dopamine system table opener (Hurley 2010 #1270 J. Boy 2000 Lee 18F-DOPA AADC PET / 11C-DTBZ / 11C-MP / Seeman-Niznik 1990 / Hisahara-Shimohama 2011); Dopamine metabolites in PD (continued); Dormant dopaminergic neurons; Summary of dopamine system
20240722_182919Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopamine system > Dopa ImagingMicrodialysis tail (Wegrzynowicz 2019), Nigrosomes (dopaminergic) neurons in SNpc (postmortem 98% loss in nigrosome-1), brain MRI swallow-tail panels, Dopa Imaging — dopamine synthesis capacity (11C-LDOPA / 18F-DOPA / Brooks / Snow / Hunhoff-Detto / Sossi / Morley / Adams / Lee / Oehme / Nagatsu narratives), dopamine transporter Imaging opener; Nigrosomes (dopaminergic) neurons in SNpc; Dopa Imaging — dopamine synthesis capacity; Dopamine transporter Imaging
20240722_182922Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopamine transporter Imaging > DATscan in PDDAT imaging products (PET / SPECT tracers / sensitivity / normal reference values), DATscan analogue references, DATscan in PD findings (caudate / putamen / striatum percent reduction; UPDRS correlation), Datscan and CSF NfL effect size, Datscan progression in PD opener (Simuni 2018 #2272 PPMI 대략 인 10%/year); DAT imaging products; DATscan in PD; Datscan and CSF NfL effect size; Datscan progression in PD; …
20240722_182926Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopamine transporter Imaging > DATscan in PDDatscan progression in PD (Simuni 2018 #2272 Table 5 baseline-Month 6/12/24/48; Table 7s Year 1/Year 4 percent change), Arthur volcano AD-PD database mining longitudinal DaT trajectories + power analysis evidence panels, Kim 2018 review Table 1 Previous Longitudinal Studies of DAT Imaging in PD Patients (Marek/Seibyl references), Correlation between Datscan & MDS-UPDRS opener (TABLE 4B Spearman correlations); Datscan progression in PD (continued); Arthur volcano AD-PD (Simen AAA 18-Apr-2018, S pots); (Jpod review used by chajn Kim 2018 #2018); Correlation between Datscan & MDS-UPDRS; …
20240722_182929Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopamine transporter Imaging > Rodent Dopamine transporterTABLE 4C / 4A correlation tables (Year 0/1/2/4 Spearman MDS-UPDRS × DATSCAN SBR), 6Mo vs UPDRS III / 4Y vs UPDRS III longitudinal rows (Tribais 2019 / Toiraj 2022), Correlation between Datscan & histologic markers (Doplowski 2013 #1067 mice TH/FA/DAT/striatal-fiber correlations), Correlation between Datscan & SN volume (Isaacs 2016 #1065 cross sectional), Rodent Dopamine transporter imaging — Summary of imaging centers (QST / Sekirai Medical / CIIA at Kawasaki / Medi-physics / Hamamatsu Photonics), CBigD studies opener; Longitudinal correlation between Datscan and UPDRS; Correlation between Datscan & histologic markers; Correlation between Datscan & SN volume; Rodent Dopamine transporter imaging; …
20240722_182932Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopamine system > Neuromelanin pigmentRodent imaging centers overlap, KBigD studies (KH Park 2020 18F-PR-PE2I MPTP-mouse / Js 2019 11C-PE2I 6-OHDA-rat / AAV SNCA-rat), Charles Rivers studies, SWEDD section (Marek 2018 #1283 PD PROTOpos), Neuromelanin pigment definition / Expression / DISTRIBUTION / Synthesis pathway diagram (DA → DA-quinones → DA-protein adducts → Pheomelanin → Eumelanin / Neuromelanin organelle); SWEDD; PD PROTOpos (Marek, 2018 #1283); Neuromelanin pigment; definition; …
20240722_182935Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopamine system > Neuromelanin-MRINeuromelanin pigment biochemistry (Zecca review caption: NM-pigment biosynthesis & NM-containing organelle formation in human SN), Function (chelate iron / pigmented neurons vs TH correlation, Mor 2008 #1643), Rodent vs NHP, Neuromelanin-MRI section opener (Principle Sulzer 2018 #1597 / Iron in non-pigmented cells / SN pars reticulata, globus pallidus, caudate nucleus), METHODS (T1-weighted FSE / voxelwise NM-MRI / test-retest), Cassidy 2019 #1595 validation studies opener (NM-MRI vs neuromelanin tissue concentration / dopamine release / neural activity / clinical severity); Function; Rodent; Neuromelanin-MRI; Principle; …
20240722_182938Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopamine system > Neuromelanin-MRINM-MRI validation table tail (Cassidy / Isaias / Ito), Longitudinal NM-MRI in PD (Cristian Salinas 20220625 commentary; Wirth 2022 #2071 Saljas cohort 140+97; vsNMc / disNMc / vsNMs annualized decay rates, UPDRS-III correlation), 79 early PD vs 32 HV cohort with manual SN segmentation, Dopamine Receptor imaging section opener (Booth 2015 #1745 raclopride PET / Bezard 2001 #1748); Longitudinal; Cristian Salinas (20220625); Dopamine Receptor imaging
20240722_182941Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Druggability79 early PD vs 32 HV cohort tail (cleaner copy at 20240722_182938), Dopamine Receptor Imaging continued (Booth 2015 #1745 raclopride PET / Bezard 2001 #1748 MPSP Monkey ABL plots), Druggability section opener (Lipophilicity definition / CNS penetration formulas / Kpuu values for paracetamol / HDACA / MOC950 / NLRP3 inhibitor / NoeITherm / AC immune compounds / selinexor; Rat Brain/Plasma Ratio bar chart); Dopamine Receptor Imaging (continued); Druggability
20240722_182944Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Druggability > DWI vs DTIDruggability; DWI VS DTI; Uncertain Spans
20240722_182947Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Druggability > DWI vs DTI > Dyskinesia > Clinical presentation of Dyskinesia in PD > MOA of DyskinesiaDruggability (continued tail); DWI VS DTI; Dyskinesia; Clinical presentation of Dyskinesia in PD; MOA of Dyskinesia; …
20240722_182950Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Druggability > DWI vs DTI > Dyskinesia > Clinical presentation of Dyskinesia in PD > MOA of Dyskinesia > Outcome Measures Dyskinesia > Animal models of Dyskinesia > Pipeline of DyskinesiaDyskinesia; Clinical presentation of Dyskinesia in PD; MOA of Dyskinesia; Outcome Measures Dyskinesia; Animal models of Dyskinesia; …
20240722_182953Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Druggability > DWI vs DTI > Dyskinesia > Pipeline of Dyskinesia > EEG > Bands > EEG in PDPipeline of Dyskinesia (continued); EEG; Bands; EEG in PD; Uncertain Spans
20240722_182956Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > EEG > Bands > EEG in PD > Limitations > Sleep EEG > Effect sizeEEG in PD (continued); Limitations; Sleep EEG; Effect size; Uncertain Spans
20240722_182959Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > EEG > EEG in PD > Limitations > Sleep EEG > Effect sizeSleep EEG (continued); Effect size (continued); post-hoc; NLRP3 (or ASC) vs TH; correlation / Exact test; …
20240722_183002Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > EEG > Effect size > Efficacy modelling > Electrophysiology > Anatomy of a neuron > MethodsCorrelation / Exact test (continued); Efficacy modelling; Electrophysiology; Anatomy of a neuron; Methods; …
20240722_183006Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Electrophysiology > Anatomy of a neuron > Methods > Resting potential (Inactive state) > ProcessElectrophysiology; Anatomy of a neuron; Methods; Resting potential (Inactive state); Process; …
20240722_183009Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Electrophysiology > Process > voltage-dependent ion channels > ERB > Endoplasmic Reticulum (ER) > 4 ExosomeResting potential (Inactive state); Process; voltage-dependent ion channels; ERB; Endoplasmic Reticulum (ER); …
20240722_183012Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Endoplasmic Reticulum (ER) > 4 Exosome > ConsoRCC > Example > 4 NDE > neuronal exosome enrichment procedure > Oligodendrocyte-derived exosomeEndoplasmic Reticulum (ER); Exosome; function; Limitation of exosome; Process; …
20240722_183015Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Oligodendrocyte-derived exosome > Eye > Fibroblast > Cell model system for CNS? > GABA > Further readings > GABA imaging > review (Magrinelli, 2016 #1063)Oligodendrocyte-derived exosome (continued); Eye; Fibroblast; Cell model system for CNS?; GABA; …
20240722_183018Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > GABA imaging > Gait > 4 Gait > Assessment of Gait > Galectin-3 > GCS inhibitorGABA (continued); Further readings; GABA imaging; Gait; Assessment of Gait; …
20240722_183021Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Gladstone Institute > Glutamate > Imaging > Goals > FY22Gladstone Institute; Glutamate Imaging; Goals; FY22; Experimental Medicine – KPIs for 2022; …
20240722_183024Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Goals > FY22 > GWAS > Modifiers > SummaryGWAS; Modifiers; Summary; Uncertain Spans
20240722_183028Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > GWAS > Modifiers > Summary > LSD genesLSD genes and variants in the IPDGC cohort; Uncertain Spans
20240722_183031Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > GWAS > Summary > LSD genes > GPR37 > Glossary > H&Y (Hoehn and Yahr)LSD genes and variants in the IPDGC cohort; GPR37; Target; Glossary; H&Y (Hoehn and Yahr); …
20240722_183034Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > GPR37 > Glossary > H&Y (Hoehn and Yahr)GPR37; Target; Glossary; H&Y (Hoehn and Yahr); Uncertain Spans
20240722_183037Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > H&Y (Hoehn and Yahr) > HA-tag > Hearing lossHA-tag; Hearing loss; Nigrosome1 MRI; Nigrosome1 MRI; Uncertain Spans
20240722_183040Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Hearing loss > Heuron > Hirozane, Yoshihiko > HR Performance Review (including 360)Heuron; Hirozane, Yoshihiko; HR Performance Review (including 360); Human Biological Samples; Uncertain Spans
20240722_183044Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > HR Performance Review (including 360) > Human Biological Samples > Imaging > PETHuman Biological Samples; Imaging; PET; Immunity; Uncertain Spans
20240722_183047Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Immunity > Autoimmunity (Autoantibody) > Evidence > ImmunoPET > ImnewrunImmunity; Autoimmunity (Autoantibody); Evidence; ImmunoPET; Imnewrun; …
20240722_183050Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Inflammation > ALS > aSyn (therapeutic) antibody > ComorbidityaSyn (therapeutic) antibody; Comorbidity; Summary (sento M); Uncertain Spans
20240722_183055Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Comorbidity > Signals > Inflammasome > In vitro model of neuroinflammationSignals; Inflammasome; In vitro model of neuroinflammation; Uncertain Spans
20240722_183058Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Inflammasome > In vitro model of neuroinflammation > Imaging neuroinflammationImaging neuroinflammation; Uncertain Spans
20240722_183101Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Inflammasome > In vitro model of neuroinflammation > Imaging neuroinflammationUncertain Spans
20240722_183115Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Inflammasome > In vitro model of neuroinflammation > Imaging neuroinflammation > Structure of microglia > Phenotypes of microglia > Markers of microgliaMarkers of microglia; Priming of MG; Receptors on microglia; Uncertain Spans