Litcius/Paper detail

Food and Drug Administration Lifts Clinical Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Linked to Patient Death

Alex Philippidis

2022Human Gene Therapy30 citationsDOI

Topics & Concepts

Duchenne muscular dystrophyFood and drug administrationMedicineMuscular dystrophyGenetic enhancementDrugGeneOrphan drugPharmacologyBioinformaticsInternal medicineGeneticsBiologyCRISPR and Genetic EngineeringCAR-T cell therapy researchVirus-based gene therapy research