In an opinion article, investigators argue that gene therapies may not achieve their full potential if the innate inflammation in Duchenne muscular dystrophy is not effectively managed.

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

The MESA data demonstrated sustained disease stabilization, reinforcing prior ARCH and CANYON findings. Importantly, CANYON ...

The dad of a tragic Scots boy has called for more help for families with terminally ill children - after selling their home ...

Elevidys has been given full approval to treat ambulatory patients with DMD, with an accelerated approval in non-ambulatory ...

New consensus guidelines outline best practices for orthopedic management in Duchenne muscular dystrophy, focusing on ...

Ifetroban improved heart function and reduced cardiac damage biomarkers in people with DMD in a Phase 2 clinical trial.

Capricor stock drops as FDA cancels advisory meeting on Duchenne therapy; leadership changes and regulatory tensions fuel ...

The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...

Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...

The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...

FDA has indicated that an Advisory Committee meeting is not required at this time In-person late-cycle review meeting scheduled for mid-July Biologics License Application (BLA) remains under Priority ...