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First Duchenne gene therapy approved by FDA for young children

BioPharma

United statesSusan finazzoPeter marksCelia wittenJennifer handtDoug ingramJerry mendellLisa leeJohn brandsemaTim revellDrug administration onDrug administrationSarepta therapeuticsNationwide childrenVirginia techParent project muscular dystrophy

FDA panel narrowly backs approval of Sarepta s Duchenne gene therapy

BioPharma

United statesAnthony amatoLisa leeMelanie hennickEmmanuel adu gyamfiPeter marksCaleb alexanderSteven pavlakisSusan finazzoCelia wittenDrug administrationHealth sciences universitySarepta therapeuticsBiopharma diveStat newsSciences university

Gene therapy for muscular dystrophy stirs hopes and controversy

The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.

University of floridaUnited statesYale schoolUnited kingdomBarry byrneSusan finazzoChris finazzowereDouglas ingramReshma ramachandranGlen nuckollsDebra millerDrug administrationMuscular dystrophy associationNational institute of neurological disordersYale school of medicineChase finazzo

FDA considers new muscular dystrophy treatment using experimental gene therapy : Shots

The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.

United kingdomUniversity of floridaUnited statesYale schoolChris finazzoBarry byrneDouglas ingramSusan finazzoReshma ramachandranChris finazzowereRosa reyes susan finazzoDebra millerGlen nuckollsNational institute of neurological disordersMuscular dystrophy associationDrug administration

Gene therapy for muscular dystrophy stirs hopes and controversy

The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.

Yale schoolUnited statesUnited kingdomUniversity of floridaBarry byrneChris finazzowereReshma ramachandranGlen nuckollsDebra millerSusan finazzoDouglas ingramDrug administrationMuscular dystrophy associationYale school of medicineNational institute of neurological disordersChase finazzo

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