hd regulatory science consortium (hd-rsc) · hd regulatory science consortium (hd-rsc) a consortium...
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HDRegulatoryScienceConsortium(HD-RSC)AconsortiumaimedatacceleratingtreatmentsforHuntington’sdisease
Kick-OffMeeting,SheratonSilverSpringHotel,November6-7,2017
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Robi BlumensteinCHDIFoundation
“TheVision:CHDI’sDedicationtoSuccessfulHDTreatments”
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MarthaBrumfieldCriticalPathInstitute
“Consortia-BasedStrategiesinNeurodegenerativeDiseases:CriticalPath
Institute’sTrackRecordinCollaborativeEfforts”
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MikePanzara,WaveLifeSciencesScottSchobel,RocheLouiseVetter,HDSA
JulianaBronzova,UniQureMarkGordon,Teva
“TheNeed:WhataretheKeyChallengesforDrugDevelopmentinHD”
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Moderator:JeffCarroll,WesternWashingtonUniversity
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BillyDunnFDA
“RegulatoryImpactforHuntington’sDisease:FDAPerspectives”
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10:35AM– 10:50AMBREAK
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ManuelHaasEMA
“RegulatoryImpactforHuntington’sDisease:EMAPerspectives”
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Issam ZinehFDA
“ModelInformedDrugDevelopment”
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BrianCorriganPfizer
“FromModel-BasedClinicalTrialEnrichmenttoComprehensiveClinicalTrialSimulation”
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11:45AM– 12:30PM
LUNCH
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AmritaMohanCHDIFoundation
“SuccessinSharingDatafromHDNaturalHistoryStudies”
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BrianCorrigan,PfizerBernhardLandwehrmeyer,UlmUniversity
KevinKrudys,FDAGeraldPodskalny,FDA
CharlesVenuto,UniversityofRochester
“HDDiseaseProgressionModeling:AligningDatawithContextofUseApplications”
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Moderator:KlausRomero,CriticalPathInstitute
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1:30PM– 1:45PM
BREAK
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CristinaSampaioCHDIFoundation
“RationaleandImpactofBuildingaComprehensiveHDClinicalDatabase”
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EricSiemersEliLilly
“BiomarkersasToolstoEnableDecision-MakinginHDDrugDevelopment”
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GlennStebbinsRushUniversity
“ClinicalOutcomeMeasuresinHD:BeyondUHDRS”
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SteveHersch,VoyagerBernardRavina,Voyager
BillyDunn,FDAEricBastings,FDA
“OutliningaRoadmapforClinicalTrialsinPre-ManifestHD”
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Moderator:KarlKieburtz,UniversityofRochester
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3:45PM– 4:00PMBREAK
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CharlesSabinePatientAdvocate
“TheImpact:WhyThisMatterstoPatients”
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6:00PMDINNERat:
CopperCanyonGrill928EllsworthDriveSilverSpring,MD
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EmilyGantmanCHDIFoundation
“SummaryandHighlightsfromDay1ObjectivesforDay2”
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DianeStephenson&DebraHannaCriticalPathInstitute
“TheOperations:HowHD-RSCWillWork”
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Q&A23
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DianeStephensonCriticalPathInstitute
IntroductiontoBreakoutSessions
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10:00AM– 10:15AM
BREAK
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10:15AM– 12:00PMBREAKOUTSESSIONS
1)Model-basedStrategiesforClinicalTrailEnrichment:FocusonBiomarkersRoom:Magnolia
2)ClinicalOutcomeAssessmentMeasuresinHDforUseinTrialsRoom:Persimmon1
3)TherapeuticDevelopmentinPre-manifestHD:OpportunitiesandChallengesRoom:Persimmon2
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CristinaSampaioCHDIFoundation
“MeetingSummaryandConclusions”
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