First, she mentioned the 2016 approval of eteplirsen (Exondys 51; Sarepta), which was the first gene therapy for DMD but provoked ... with 4 copies of the SMN2 gene, who were being wrongly classed ...

Duchenne is caused by mutations of the DMD gene, which affects the production of the muscle ... Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by ...

DMD Long-term functional outcomes ... Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...

Sarepta Therapeutics said it will update its prescribing information for Elevidys® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...

Wave Life Sciences Ltd. plans to file with the U.S. FDA for accelerated approval of WVE-N531, an exon skipping ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

Suffolk County Police Department A New York woman has been arrested for allegedly posing as a dentist and performing "complex dental procedures" without a license, per local authorities.

Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy (DMD), becoming the first non-steroidal option for patients with all ...