On a recent summer morning off the coast of Bat Yam, south of Tel Aviv, a dozen Israeli volunteers in wetsuits splashed in the sparkling blue waters of the Mediterranean Sea. But they weren’t there just for fun. The focus of their attention: three young boys gripping specially modified surfboards…
Move over, Sarepta Therapeutics, Solid Biosciences, PTC Therapeutics and Pfizer. Vertex Pharmaceuticals, which dominates the cystic fibrosis drug market, has just entered the multibillion-dollar race to find effective treatments — and maybe even cures — for two other rare diseases: Duchenne muscular dystrophy…
Aiming to enhance patients’ lives, a team of University of Manitoba biomedical students has produced autonomous designs — including a wheelchair hand warmer for those living with muscular dystrophy (MD). The overarching goal during the University of Manitoba Biomedical Engineering Design (BMED) Team’s first year was…
Years ago, doctors believed that no exercise was best for boys with Duchenne muscular dystrophy (DMD). Today, DMD experts all agree: exercise extends lives. But what kind of exercise is ideal for Duchenne kids? How much is too much? And what about stretching, night splints and specialized equipment? Physical…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…
The ongoing Phase 2 HOPE-2 study has been cleared to continue exploring the potential of CAP-1002 therapy in boys and young men with advanced Duchenne muscular dystrophy (DMD). An independent Data and Safety Monitoring Board (DSMB) made the decision after its thorough safety and futility review and the…
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
Climbing up the world’s tallest mountain isn’t for everyone. With that in mind, a small New England-based charity plans just such an adventure this November — for the third time, no less — to raise at least $100,000 to help cure Duchenne muscular dystrophy (DMD). Tonya Dreher is executive…
Invitae has opened a program offering free genetic testing and post-test counseling to people suspected of having diseases that include muscular dystrophy (MD) under a partnership with pharmaceutical firms. The goal is to promote early diagnosis and treatment. The initiative, called Detect, targets disorders for which testing…
Filippo Buccella, an Italian pharmacist from Rome, clearly remembers the day in 1993 when he brought his nearly 3-year-old son, Luca, to the hospital. Luca was about to undergo surgery for his adenoids when a test revealed that his creatine kinase level was over 11,000 — a hallmark symptom of…
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