PGN-EDO53, an experimental therapy designed to treat Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping, showed…
Articles by Marisa Wexler, MS
Sarepta Therapeutics plans to begin a clinical trial to test the antibody-cleaving therapy imlifidase as a pre-treatment for SRP-9001…
Santhera and ReveraGen have completed their rolling application asking the U.S. Food and Drug Administration (FDA) to approve the dissociative…
Treatment with Viltepso (viltolarsen) helps preserve motor function in boys with Duchenne muscular dystrophy (DMD) amenable to…
The U.K.’s National Institute for Health and Care Excellence (NICE) has published new draft guidance that does not recommend…
Sarepta Therapeutics has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to grant…
Four months of treatment with the experimental oral therapy EDG-5506 reduced markers of muscle damage and improved measures of physical…
For the fourth year, a family in Montana is hosting “Calves to Cure,” a cattle auction that aims to…
Under an agreement with the Broad Institute, Sarepta Therapeutics has acquired an exclusive license to the gene therapy…
Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant accelerated approval to the company’s…