Posted by Michael Wonder on 17 May 2018
Myonexus Therapeutics receives FDA rare paediatric drug designation for pioneering treatment of limb girdle muscular dystrophy Type 2E
16 May 2018 - Rare paediatric disease designation for MYO-101 program reflects compelling data and enables priority eeview voucher eligibility.
Myonexus Therapeutics and Nationwide Children’s Hospital announced today that the U.S. FDA has granted rare paediatric disease designation for the MYO-101 program, an AAV-based gene therapy for the treatment of limb girdle muscular dystrophy type 2E.
In February 2018, the MYO-101 program was granted orphan drug designation by FDA.
