Independent living can be achievable for many adults with muscular dystrophy (MD), but there are various factors to consider to ensure you get the best care and help available. Often the greatest challenge is finding and managing your personal care assistant (PCA), but according to the Muscular…
A U.S. Food and Drug Administration (FDA) advisory board concluded that evidence supporting the effectiveness of Translarna (ataluren), by PTC Therapeutics, as a treatment for Duchenne muscular dystrophy is inconclusive. The opinion, which was endorsed by 10 of the 11 advisory board members at its Sept. 28 meeting, also stated that…
The newly founded Collaborative Trajectory Analysis Project (cTAP) coalition of stakeholders has invited Summit Therapeutics, a company specialized in the discovery and development of drugs targeting Duchenne muscular dystrophy (DMD), to support the development of new therapeutic products aiming to treat DMD, such as utrophin modulators. cTAP…
In this video from Muscular Dystrophy UK, two teenagers share what it’s like living with the disease and some of the frustrations they experience on a day-to-day basis. MORE: How service dogs can benefit people with neuromuscular disorders Fourteen-year-old Sam from Cheltenham and 13-year-old Zoe…
In this video from Muscular Dystrophy UK, Dr. Ros Quinlivan from the Queen’s Square Centre for Neuromuscular Diseases in London talks about health care for people living with congenital muscular dystrophy. MORE: Four treatment areas for muscular dystrophy Dr. Quinlivan explains that CMD covers a wide…
The Muscular Dystrophy Association (MDA) has launched its 2017 “Live Unlimited” awareness and fundraising campaign. Running until Oct. 11, it targets families affected by muscular dystrophy (MD) and other neuromuscular diseases, and aims to help patients pursue their personal and professional goals. This year’s campaign features inspiring examples:…
Olympian and fastest man on the earth, Usain Bolt, has teamed up with watch makers Hublot to design a special edition watch that will be auctioned in support of Duchenne muscular dystrophy (DMD) research, according to TMZ.com. MORE: Six complications of muscular dystrophy The 18-carat gold and sapphire watch is…
The nonprofit group CureDuchenne won $50,000 at a recent Shark Tank-like competition in Irvine, California, to pitch scientific projects. The “RARE Battle of the Brains” competition, held Sept. 14, was organized by Scientist.com, a marketplace for scientific services, and Global Genes, a rare disease patient advocacy group. CureDuchenne won the…
Synpromics and Solid Biosciences have established a collaborative agreement to develop new potential gene therapies for the treatment of Duchenne muscular dystrophy (DMD). According to the partnership terms, Solid Biosciences will gain access to muscle-selective promoter candidates developed by Synpromics, which are…
Muscular dystrophy (MD) is a neuromuscular disorder, characterized mainly by progressive muscle loss. Physical therapy plays an important role in MD treatment, however, there are many myths out there about the practice and its benefits. The American Physiotherapy Association has useful information to help set the record straight. You need a referral…
Get regular updates to your inbox.