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 ...