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Defining Meaningful Outcomes In Gene Therapy Trials For Duchenne Muscular Dystrophy

Published: February 28, 2022

Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is an X-linked recessive disease that affects approximately one in...

Source: clinicalleader.com

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Roxana Donisa DreghiciPatrick GonzalezCarl MorrisCarla MorrisJ Patrick GonzalezDalle PazzeUniversity Of MedicineFranklin Pierce CollegeSyracuse UniversityBoston University School Of MedicineDepartment Of PhysiologyResearch UnitPfizer Rare Disease Research UnitRutgers UniversityEuropean Medicines AgencyUniversity Of PennsylvaniaNovo NordiskSolid BiosciencesNorth Star Ambulatory AssessmentUpper LimbSolid Bioscience PhasePediatric Outcomes Data Collection InstrumentDuchenne Video AssessmentChief Scientific OfficerRare Disease Research Unit