W. Zhang

Safety and Efficacy of Delandistrogene Moxeparvovec versus Placebo in Duchenne Muscular Dystrophy (EMBARK): Pivotal Phase 3 Primary Results

I. Seelmann, Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald et autres

Background/Purpose: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations. Delandistrogene moxeparvovec is an adeno-associated viral vector-based gene transfer therapy, designed to compensate for absent functional dystrophin in DMD by delivering a transgene encoding engineered micro-dystrophin, which retains key functional wild-type …

de, us, gb, it, jp, es, ch (code pays fourni par la source)

P24 Safety and tolerability of Eteplirsen in patients 6–48 Months old with DMD amenable to exon 51 skipping: an open-label extension study

E. Mercuri, A. Seferian, Nicolas Deconinck, L. Orogun et autres

it, fr, be, us, gb (code pays fourni par la source)

Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping

Eugenio Maria Mercuri, A. Seferian, Laurent Servais, Nicolas Deconinck et autres

Eteplirsen is FDA-approved for the treatment of Duchenne muscular dystrophy (DMD) in exon 51 skip-amenable patients. Previous studies in boys > 4 years of age indicate eteplirsen is well tolerated and attenuates pulmonary and ambulatory decline compared with matched natural history cohorts. …

it, fr, be, gb, us (code pays fourni par la source)