FDA awards LAMA2-CMD therapy MDL-101 orphan drug status

MDL-101, Modalis Therapeutics’ epigenetic editing therapy for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The designation is intended to encourage the development of therapies for serious or life-threatening rare diseases, which are those affecting fewer than…

Rare pediatric disease designation granted to LAMA2-CMD therapy

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to MDL-101, an epigenetic editing therapy that Modalis Therapeutics is developing for LAMA2-related congenital muscular dystrophy (LAMA2-CMD) — a genetic condition that’s characterized by muscle wasting evident at birth or shortly thereafter. This FDA status…

Diabetes Treatment, Metformin, Seen to Aid Muscle Function and Overall Health of Mice with Congenital Muscular Dystrophy

Metformin, a diabetes treatment, eased disease progression and improved muscle function in female — and, to a lesser extent, in male — mice altered to have congenital muscular dystrophy with laminin α2 chain-deficiency (LAMA2-CMD), a study reports. Its researchers also reported that this type of congenital muscular dystrophy — the second…