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

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 ...

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.