Columnist Betty Vertin, who has seven children, tries to organize the chaos in her family's lives, though no week is typical.

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 ...

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 ...

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

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 ...

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.

Capricor Therapeutics will soon begin filing an application seeking U.S. approval of its cell therapy deramiocel for cardiomyopathy, a disease of the heart’s muscle, in people with Duchenne muscular ...