Clinical examination was normal in most patients, muscle hypertrophy ... had Becker muscular dystrophy. Progressive muscle wasting and limb girdle weakness was seen in one patient with homozygous ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular ...

to treat Duchenne muscular dystrophy (DMD) in patients aged 6 years and older who can walk. Conditional marketing authorization is granted when the benefit of immediate availability outweighs the ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular ...

Local intramuscular administration of the antisense oligonucleotide PRO051 in patients with Duchenne's muscular dystrophy with relevant mutations was previously reported to induce the skipping of ...

Duchenne muscular dystrophy (DMD), recognised by the World Health Organization (WHO) as a rare disease, is the most common form of childhood muscular dystrophy. It is a progressive genetic ...

Cas12Max-based gene editing therapy for Duchenne muscular dystrophy, one of the most severe forms of the inherited muscular dystrophies that affects primarily boys. Huidagene Therapeutics and ...

A new study led by researchers at Hospital for Special Surgery (HSS) suggests that currently available therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy (DMD ...