FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys
The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics gene therapy for Duchenne...
The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys
Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on...
Brazil Suspends Use of ELEVIDYS (delandistrogene moxeparvovec)
The Brazilian Health Regulatory Agency (Anvisa) announced that it has temporarily suspended the marketing, distribution, manufacturing, import, advertising, and use of the gene therapy...
FDA Designates Avidity Biosciences’ Delpacibart Zotadirsen (del-zota) as a Breakthrough Therapy for the Treatment of DMD in Individuals with Mutations That Allow for Exon...
Avidity Biosciences today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota) for the treatment...
The FDA Designates PBGENE-DMD as an Orphan Drug by Precision BioSciences to Treat Duchenne Muscular Dystrophy
Precision BioSciences, a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with...