PTC Therapeutics shared additional data from a Phase 3 trial of Translarna (ataluren), showing that the treatment slightly slowed disease progression in ambulatory boys with Duchenne muscular dystrophy. The data was presented at the recent American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting in Phoenix, Arizona.
This Muscular Dystrophy UK video is all about Robert Watson from Renfrew, Scotland. The 29-year-old was diagnosed with Duchenne muscular dystrophy 23 years ago, when he was just six years old. In his spare time, Robert likes to play powerchair football and socialize with his friends at nightclubs, concerts…
Phrixus Pharmaceuticals and several Duchenne muscular dystrophy (DMD) organizations will jointly conduct the first open-label trial of Poloxamer-188 NF, the active ingredient in Carmeseal-MD, to treat DMD in patients who can no longer walk. The trial, to take place at Cincinnati Children’s Hospital, will evaluate the drug’s performance on respiratory…
https://www.youtube.com/watch?v=9z4jkn4Wv3w In this video from MoveForwardPT, a physical therapist (PT) from the Children’s National Medical Center talks about the role PTs play in optimizing movement for children with muscular dystrophy (MD) and spinal muscular atrophy (SMA). MORE: The cost…
For the ninth year in a row, “Coach to Cure MD” and Parent Project Muscular Dystrophy (PPMD) will mark Duchenne Awareness Month  by using college football games to raise awareness of Duchenne muscular dystrophy (DMD) and money to promote research into treatments and a possible cure. The effort, a partnership between the…
The agency that plays the key role in European Union therapy authorizations has recommended against approval of Raxone (idebenone) as a Duchenne muscular dystrophy (DMD) treatment. Santhera said in a press release that it plans to appeal the Committee for Medicinal Products for Human Use’s decision, which covers Raxone in DMD patients not being treated…
It’s not always easy to be positive with a disability, especially in the beginning. It’s hard to see your friends do things that you can’t. The stares are hard to ignore, and there are people who simply don’t want to understand your situation. It doesn’t make life any easier, but…
In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial of a new gene therapy for Duchenne’s muscular dystrophy (DMD). Final go-ahead for the trial awaits U.S. Food and Drug Administration (FDA) approval of an Investigational New Drug (IND) application made for…
These five muscular dystrophy patients are inspiring the entire community with their stories and their outlook on life. In this inspiring video from MDA, we meet Jimmy Valdes and his wife, Julie. Originally from Queens, New York, 47-year-old Jimmy was diagnosed with spinal muscular dystrophy (SMA) in 1971…
Managing a chronic illness can be difficult. There are many different medications to take (often at different times), appointments to remember, symptoms to keep track of, and lots of information to absorb. Thankfully, living in a digital age means that there are numerous mobile apps that can help you manage…
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