Agamree (vamorolone) was recommended for use in the NHS in England, Wales, and Northern Ireland for treating Duchenne ...

FSHD exacts a toll on columnist Robin Stemple when he is out and about. He calls these symptom flares "FSHD hangovers." ...

Eight $250,000 prizes will be awarded for key milestones in FSHD treatments and $8 million will be given for showing clinical ...

Arrakis' oral therapies targeting disease-driving RNA in myotonic dystrophy have shown promise in preclinical studies.

Columnist Shalom Lim describes a moving performance at the Enabling Lives Festival in Singapore, an example of the power of ...

Sarepta has acquired the global rights to develop ARO-DUX4 and ARO-DM1, two RNAi therapeutic candidates for types of muscular ...

Couch shopping made columnist Betty Vertin discover she was focused on her three children with DMD rather than the rest of her family.

The case is reported of a young woman who began having DMD symptoms before getting pregnant, allowing her to receive genetic ...