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

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

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

Leerink Partners analysts pointed out in a note this morning that this 3.8-second improvement is greater than the 2.7-second benefit seen one year after taking Sarepta’s DMD gene therapy Elevidys.

Regenxbio (RGNX) stock gains and Solid Biosciences (SLDB) stock falls after Sarepta Therapeutics' (SRPT) safety update on ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients. DMD is a form of ...

Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta ...

The virus isn't harmful, but the gene it's carrying is known to put the brakes on the progression of DMD. The hope is that this therapy slows down Colton Belluzzo's disease, buying time for him to ...

US-based CureDuchenne has formed a partnership with Children's Hospital of Orange County (CHOC) to launch a new clinic for patients with Duchenne muscular dystrophy (DMD). This collaboration aims ...

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for ...

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