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 ...

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

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