Duchenne muscular dystrophy

News

Taiho has suffered a blow to its near-term pipeline after a drug candidate for Duchenne muscular dystrophy (DMD) missed the mark in a phase 3 trial.

News

The FDA has launched a formal investigation into two liver failure-related deaths in patients treated with Duchenne gene therapy Elevidys.

News

Another death from liver failure has been seen with Roche and Sarepta's Duchenne muscular dystrophy therapy Elevidys, prompting a pause on treatment.

News

Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular dystrophy (DMD).

News

Three studies of Sarepta's Elevidys Duchenne gene therapy have been placed on hold as the death of a 16-year-old boy is investigated.

News

The death of a patient treated with Sarepta's Duchenne muscular dystrophy gene therapy Elevidys has hit its share price.