High Anti-AAV Antibodies May No Longer Be a Barrier to Elevidys Administration
One of the most significant obstacles to the application of Elevidys gene therapy developed for Duchenne muscular dystrophy disease is the high anti-AAV antibodies....
MetrioPharm’s MP1032 Receives EMA Orphan Drug Designation in Duchenne Muscular Dystrophy
MetrioPharm announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD) to its lead compound, MP1032 for the treatment of children...
FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’
Elevidys, a gene therapy for Duchenne Muscular Dystrophy (DMD) from Sarepta Therapeutics, is now available to ambulatory patients, and the U.S. Food and Drug...
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...