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Defining Meaningful Outcomes In Gene Therapy Trials For Duchenne Muscular Dystrophy

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

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 pennsylvania

Solid Biosciences Reports 1 5-Year Data from Patients in the Ongoing IGNITE DMD Phase I/II

Solid Biosciences Reports 1 5-Year Data from Patients in the Ongoing IGNITE DMD Phase I/II
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United statesUnited kingdomIlan ganotVamshi raoJeffrey chamberlainDongsheng duanDavid careyErich sandovalCaitlin lowieNorthwestern university feinberg school of medicineUniversity of missouriExchange commissionUniversity of washingtonEuropean unionSolid biosciences incWorld muscle society

Solid Biosciences Reports Efficacy and Safety Data from the Ongoing IGNITE DMD Clinical Trial and Resumption of Patient Dosing in the 2E14 vg/kg Cohort

Solid Biosciences Reports Efficacy and Safety Data from the Ongoing IGNITE DMD Clinical Trial and Resumption of Patient Dosing in the 2E14 vg/kg Cohort
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United statesUnited kingdomBarry byrneDongsheng duanDavid careyCraigm mcdonaldIlan ganotValeria ricottiJeffrey chamberlainErich sandovalPowell gene therapy centerUniversity of californiaUniversity of floridaUniversity of rochester medical centerUniversity of missouriUniversity of washington