Pediatrics

FDA Grants Orphan Drug Status to MDL-101 for LAMA2-CMD Treatment Modalis Therapeutics Corporation has received Orphan Drug Designation (ODD) from the FDA for MDL-101, its promising treatment for congenital muscular dystrophy type 1A (LAMA2-CMD). This designation is a significant step, aimed at fostering the development of essential therapies for rare diseases affecting fewer than 200,000 individuals in the U.S. It supports Modalis’ commitment to addressing urgent, underserved medical needs. Haru Morita, CEO of Modalis Therapeutics, expressed optimism about the development: “Receiving Orphan Drug Designation from the FDA is not only a validation of the pressing ...