Taiho Pharmaceutical has suffered a blow to its near-term pipeline after a drug candidate for Duchenne muscular dystrophy ...

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement: ...

More than 30 types of muscular dystrophy have been identified. They all cause problems with movement due to genetic mutations.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced a strategic restructuring plan ...

Sarepta Therapeutics, Inc. faces setbacks with Elevidys, safety issues, and workforce cuts, raising concerns about its ...

Del-zota is designed for patients with mutations amenable to exon 44 skipping, which is just 7% of the overall DMD patient ...

The grandmother of a four-year-old boy from Co Kilkenny diagnosed with a life-limiting genetic disorder has vowed to keep ...

This committee was dissolved only days before members were expected to add two additional rare diseases - metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD) - to what's ...

As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...