Myonexus Therapeutics receives FDA Rare Pediatric Disease Designation

(CincyTech) Myonexus Therapeutics, a clinical-stage gene therapy company developing first ever corrective gene therapies for limb girdle muscular dystrophies, and Nationwide Children's Hospital announced today that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for the MYO-101 program, an AAV-based gene therapy for the treatment of limb girdle muscular dystrophy (LGMD) type 2E. In February 2018, the MYO-101 program was granted Orphan Drug Designation (ODD) by FDA.