Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

Gene therapies, exon-skipping drugs and next-gen steroids are reshaping Duchenne muscular dystrophy care, targeting broader ages and mutations. We are witnessing the fastest pivot in rare-disease ...

In the U.S., ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. Acute serious liver injury has been observed with ELEVIDYS ...

Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) ...

In the U.S., ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...

With FDA platform technology designation for its viral vector, SRPT can fast-track other therapies using the same ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...

“For nearly a decade, Sarepta has been a leader in advancing the treatment of Duchenne muscular dystrophy through innovative treatment options. With the approval of ELEVIDYS in Japan, children ...

In the U.S., ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. The DMD indication in non-ambulatory patients is approved under ...