Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to ...

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...

A senior official at the US Food and Drug Administration has suddenly resigned after only three months in the job. Vinay Prasad’s departure followed controversy over a treatment for Duchenne muscular ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD). WVE ...

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy fell short of its main Phase 3 goal, but the firm contends the full body of evidence supports expanding the therapy’s label to ...

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...