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 ...

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

Working to better understand the concept of ableism, columnist Robin Stemple reflects on examples from his own life with ...

Setting goals, adapting, and advocating for himself are all key to columnist Patrick Moeschen's approach to life with ...

DMD treatment SMT-01, a cell replacement therapy, received FDA orphan drug and rare pediatric disease designations.

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 ...

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 ...

Rob Stemple is a lifelong advocate for people with disabilities. He was diagnosed with FSHD in 1971 at age 14. Rob struggled with its’ affects for over 50 years. He lost his eyesight in a devastating ...

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.