The symposium took place on 18 th March 2025, as part of the Muscular Dystrophy Association (MDA) Clinical & Scientific ...

It’s a devastating diagnosis that changes a child’s life — and their family’s — forever. Duchenne Muscular Dystrophy, or DMD, is a rare and fatal genetic disease that primarily affects boys, slowly ...

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 ...

However, in limb-girdle or facioscapulohumeral dystrophy little or no progression of weakness ... that the underlying molecular defect in Duchenne muscular dystrophy was affected by physical ...

Recommendation is based on Phase 3 EPIDYS trial data that demonstrated Duvyzat (givinostat) provides statistically and ...

Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular ...

Families say it's 'completely frustrating' that children with Duchenne Muscular Dystrophy are being denied access to free ...

Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular dystrophy ... showed slower disease progression over 18 months of ...