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410P GNT0004, Genethon's AAV8 vector-delivered microdystrophin gene therapy of Duchenne muscular dystrophy, first data of the phase I/II part of the GNT-016-MDYF all-in-one clinical trial in ambulant boys

Vincent Laugel, S. De Lucia, Jean‐Baptiste Davion, Nathalie Danièle, Feng Cao, Miguel Á. Sanz, Laurine Buscara, S. Blaie, L Thibaut, M.-F. Sagot, Aart Brutel de la Rivière, E. Creoff, M. Lelait, Amy M. Valent, G. Perret, S. Braun, F. Muntoni

2024Neuromuscular Disorders13 citationsDOI

Topics & Concepts

Duchenne muscular dystrophyGenetic enhancementClinical trialMedicineMuscular dystrophyPhysical therapyPediatricsGeneInternal medicineGeneticsBiologyMuscle Physiology and DisordersVirus-based gene therapy researchBiotin and Related Studies
410P GNT0004, Genethon's AAV8 vector-delivered microdystrophin gene therapy of Duchenne muscular dystrophy, first data of the phase I/II part of the GNT-016-MDYF all-in-one clinical trial in ambulant boys | Litcius