The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

After Sarepta reported the death of a patient who had recently taken the gene therapy Elevydis, patient advocacy group Parent ...

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on rare diseases, today announced that results from its Phase 2 FIGHT DMD clinical ...

The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Sarepta Therapeutics Inc.’s stock was down 21.6% in premarket trades on Tuesday after the commercial-stage biopharmaceutical company said a patient died from liver failure after using its drug ...

While data on functional endpoints are still to come, Avidity Biosciences Inc. executives said the firm is moving ahead with ...

DMD is a devastating genetic disorder that leads ... Notable findings include: -- Patients treated with deramiocel showed a reduction in disease progression, with the annual decline in PUL 2.0 ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the ...