Lindsey Shapiro, PhD,  science writer—

The U.S. Food and Drug Administration (FDA) has given the go-ahead for the first-in-human dosing of Cure Rare Disease’s (CRD) investigational genome-editing therapeutic for Duchenne muscular dystrophy (DMD). Dosing of a single patient with the treatment, dubbed CRD-TMH-001, will happen soon at the University of Massachusetts Chan Medical…

Treatment with dystrophin expressing chimeric (DEC) cells was safe and led to long-term cellular and functional improvements in the heart, respiratory, and leg muscles of a mouse model of Duchenne muscular dystrophy, a study found. The findings support “future clinical application of DEC as a safer and potentially more…

The first healthy volunteer has been dosed with an investigational exon-skipping therapy, called PGN-EDO51, for Duchenne muscular dystrophy (DMD) in a Phase 1 trial, the treatment’s developer, PepGen, has announced. The single-ascending dose trial is investigating the treatment’s safety and tolerability in about 40 healthy adult males in Canada.

The Muscular Dystrophy Association (MDA) has launched its 40th annual MDA Shamrocks fundraising campaign — one of the largest St. Patrick’s Day fundraisers in the U.S. — to raise money and to increase awareness of muscular dystrophy (MD) and related neuromuscular disorders. Throughout February and March, thousands…