Update (Dec.

“As you all know, being the creative type that I am, I love do-it-yourself projects … However, I found myself in the middle of a rather unusual project, which involved a lot of creative ‘thinking outside of the box,’ and it was more than just a do-it-yourself. It was more of a ‘do it ourselves’ project. And we did it. Successfully. Here’s the story of how we helped Nick Dupree.”

Findings from a study of 516 Italian boys show a significant improvement over the last six decades in survival time in Duchenne muscular dystrophy (DMD).

A phase 1b/2a trial to test the safety and tolerability of multiple doses of the experimental drug ISIS-SMNRx in children with spinal muscular atrophy (SMA) is now open at sites in New York and Salt Lake City, with additional sites expected to open in Boston and Dallas.

Scientists funded in part by MDA have found the cause of type 2facioscapulohumeral muscular dystrophy (FSHD), a discovery that increases scientific understanding of both forms of FSHD, will improve diagnosis of the disease and could lead to treatment advances.

Results from a completed phase 2 trial of the experimental therapy NP001 in people with amyotrophic lateral sclerosis (ALS) show that in some trial participants, the drug appeared to be effective at slowing or stopping progression of the disease.

Society hasn't always appreciated the vital role fulfilled by family caregivers. It’s only been in the past 20 years that family caregivers — now more than 65 million strong — have been officially acknowledged. Every November, National Family Caregivers Month is observed in America as a time to thank, support, educate and empower these hardworking, creative and loving individuals.

Compounds targeting exon 52 and exon 55 of the dystrophin gene as a way to treat some forms of Duchenne muscular dystrophy (DMD) will be moved into clinical trials as soon as possible, says Dutch biotechnology company