The CHMP recommends conditional marketing authorization for Duvyzat to treat Duchenne muscular dystrophy in patients aged 6 ...

A new study led by researchers at Hospital for Special Surgery (HSS) suggests that currently available therapies may help ...

A new study led by researchers at the Hospital for Special Surgery (HSS) suggests that currently available therapies may help ...

Gene therapy has emerged as a promising approach to previously untreatable conditions, including Duchenne muscular dystrophy (DMD), a progressive disorder caused by DMD mutations that leads to early ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...

Pfizer has axed trials of its domagrozumab antibody for Duchenne muscular dystrophy (DMD), after it ... is involved in the regulation of skeletal muscle growth. The decision to discontinue trials ...

On the call from BioMarin management today are Alexander Hardy, President and Chief Executive Officer; Brian Mueller, Executive Vice President, Chief Financial Officer; Cristin Hubbard, Executive Vice ...

Mutations in the dysferlin gene lead to limb girdle muscular dystrophy 2B, Miyoshi myopathy and distal ... The diagnosis was established by reduction of dysferlin in the muscle biopsy and subsequent ...

While there have been reports from drugmakers about possible interruptions with FDA matters due to staff layoffs, one company ...