News Sarepta backs down and pauses Elevidys shipments The FDA seems to have won its staring contest with Sarepta over shipments of Elevidys, as the company blinks and agrees to a pause from today.
News Sarepta resists as FDA seeks pause on Elevidys use Sarepta has defied an FDA request to halt all use of its gene therapy for Duchenne muscular dystrophy, creating an intriguing regulatory impasse.
News Taiho's late-stage Duchenne candidate fails trial 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 FDA probing deaths with DMD gene therapy Elevidys The FDA has launched a formal investigation into two liver failure-related deaths in patients treated with Duchenne gene therapy Elevidys.
News DMD gene therapy Elevidys linked to new liver failure case 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 closes on EU okay for Duchenne drug Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular dystrophy (DMD).