ATyr Pharma’s Resolaris has received orphan drug status in the United States for the treatment of limb girdle muscular dystrophy (LGMD). The Food and Drug Administration designation gives companies financial and other incentives to develop therapies for diagnosing or treating rare diseases. In the United States that means fewer…
It can sometimes be a challenge to look on the bright side when you have a chronic illness, but spending time doing things that make you happy will help keep depression at bay, help you come to terms with your illness better and begin enjoying life again. To help you feel happier,…
https://www.youtube.com/watch?v=68YO2eh1bIY YouTuber Ethan Britt takes time out from his usual filmmaking to discuss a subject close to his heart. In this video, the 18-year-old from Raleigh, North Carolina talks about when he was diagnosed with Becker muscular dystrophy (MD). Find out seven ways that Duchenne and Becker muscular…
Adults with Duchenne muscular dystrophy (DMD) may have less ability to sequentially process auditory and visual information, according to a new study. The study, “Profile of cognitive function in adults with Duchenne muscular dystrophy,” appeared in the journal Brain & Development. Most studies analyzing cognitive function in DMD focus…
CureDuchenne Ventures will give Exonics Therapeutics $5 million to advance research on a gene editing technology known as CRISPR/Cas9 that is aimed at correcting most of the mutations leading to Duchenne muscular dystrophy (DMD). DMD is a rare X-linked genetic progressive muscle disease affecting nearly 15,000 boys in the United…
Learning that a loved one has a chronic illness is devastating, and even more so if that illness is likely to cut their life short. Everyone deals with the death of loved ones differently, the grieving process is varied and there is no wrong or right way to go about…
Thirty-three years ago, doctors told Pat Furlong that her two sons, Christopher and Patrick, had Duchenne muscular dystrophy (DMD). Ten years later, the Ohio nurse — along with other fathers and mothers of Duchenne boys — founded Parent Project Muscular Dystrophy (PPMD) to raise money for Duchenne research and ultimately to…
Santhera Pharmaceuticals is recruiting patients for a new Phase 3 clinical trial evaluating its lead candidate Raxone (idebenone) in slowing respiratory decline in Duchenne muscular dystrophy (DMD) patients being treated with glucocorticoids. Raxone is an experimental drug designed to improve energy production in the mitochondria (the cell’s powerhouse) of muscle cells. In…
Researchers have developed a new method that measures the effectiveness of a cleaning process that rids defective RNA from cells. But in muscular dystrophy (MD), an imperfect RNA is better than none at all in the production of protein, which is needed for muscle function. Now researchers say they have identified a…
Having muscular dystrophy (MD) doesn’t mean that you can’t participate in fun events, you just need a little bit of imagination and some help along the way to make it happen. According to a report from the Cairns Post, a 10-year-old boy with muscular dystrophy took part in a…
Get regular updates to your inbox.