Posted by Michael Wonder on 01 Oct 2024
FDA grants rare paediatric disease designation to MDL-101 for the treatment of congenital muscular dystrophy type 1a
30 September 2024 - Modalis Therapeutics today announced that the US FDA has granted rare paediatric disease designation to MDL-101, a novel precision medicine being developed for the treatment of congenital muscular dystrophy type 1a.
MDL-101 is an experimental, epigenetic editing therapy that is being developed for the treatment of LAMA2-congenital muscular dystrophy.
