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…
I have limb-girdle muscular dystrophy type 2L (LGMD2L). My previous column, “I Live Creatively to Power Past Obstacles,” discussed how I live with my limitations. For me, the biggest challenge is navigating the world outside my home. But dealing with our disjointed healthcare system is also an issue.
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…
For several years, I’ve been feeling nauseated in the mornings after I eat breakfast. Whatever food item occupies my plate that morning — be it a chocolate chip muffin with a few strawberries or an apple with a side of yogurt — inevitably upsets my stomach. Fruits of any kind…
I believe that my physical limitations heighten my observational skills and journalistic Spidey sense. Throughout my life, I’ve realized that I notice and remember the finer details that my physically-abled friends do not. I read license plates to figure out how old a car is…
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…
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