Treatment with an RNA-based therapy approach was found to correct molecular defects in a cell model of myotonic dystrophy ...

Capricor Therapeutics started its rolling submission seeking FDA approval of its DMD therapy deramiocel for heart muscle ...

The Critical Path Institute will lead a new task force to advance the development of therapies for limb-girdle muscular ...

Columnist Patrick Moeschen has learned to tailor his descriptions of living with limb-girdle muscular dystrophy to his ...

The FDA has granted rare pediatric disease designation to Modalis' MDL-101 therapy candidate for LAMA2-related congenital ...

What’s a typical week as a caregiver of three sons with Duchenne muscular dystrophy (DMD) like? As a primary caregiver to Max, 18, Rowen, 15, and Charlie, 13, my honest answer is that I wish I knew!

The Critical Path Institute (C-Path) is launching a task force to advance the development of therapies for limb-girdle muscular dystrophy (LGMD) and will lead the efforts of its members to find a new ...

Shalom Lim is a University of Liverpool graduate, having completed an honors bachelor’s degree in criminology and security in July 2021. Born with Duchenne muscular dystrophy and diagnosed at 4 months ...

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to RAG-18, being developed as a potential treatment for both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy ...

SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). The cell replacement therapy ...