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 ...

Capricor is planning to start an application seeking the FDA's approval of deramiocel for DMD-related heart disease.

I’d hoped we had time because he’s the youngest and still walking. We’d tried so hard to buy him that time, to slow down the disease progression so that he’d have skills to save, like upper body ...

Treatment with exon 53-skipping therapy WVE-N531 led to significant increases in muscle dystrophin levels along with signs of improved muscle health and regeneration in boys with Duchenne muscular ...

I’ve been disabled for over 50 years, and I spent over 30 years working with people with disabilities. The language surrounding disability has evolved in that time. For the most part, that’s a good ...

What is BBP-418 for limb-girdle muscular dystrophy? BBP-418, or ribitol, is an oral treatment candidate designed to improve motor function in people with limb-girdle muscular dystrophy (LGMD) type 2i.