MDA Kickstart Program Receives FDA Rare Pediatric Disease Designation and Orphan Drug Designation for Congenital Myasthenic Syndrome
October 25, 2024
October 25, 2024
CHICAGO, Illinois, Oct. 25 -- The Muscular Dystrophy Association issued the following news release:
The Muscular Dystrophy Association (MDA) proudly announces that its groundbreaking MDA Kickstart program has received both an FDA Rare Pediatric Disease designation and Orphan Drug Designation for congenital myasthenic syndrome caused by CHAT (choline acetyltransferase) gene deficiency. This major milestone helps to incentivize and support the development of gene therapies targeting u . . .
The Muscular Dystrophy Association (MDA) proudly announces that its groundbreaking MDA Kickstart program has received both an FDA Rare Pediatric Disease designation and Orphan Drug Designation for congenital myasthenic syndrome caused by CHAT (choline acetyltransferase) gene deficiency. This major milestone helps to incentivize and support the development of gene therapies targeting u . . .