WVE-210201, an exon 51 skipping therapy, has shown positive safety and tolerability results in a Phase 1 clinical trial in boys with Duchenne muscular dystrophy (DMD), Wave Life Sciences announced. According to Wave Life Sciences, the positive safety data supports moving the therapy into a Phase 2/3 trial, planned to start…
At 21, Hawken Miller certainly doesn’t look like a typical Duchenne muscular dystrophy patient. For one thing, unlike most boys and young men with Duchenne, Hawken isn’t obese — or even overweight — even though he’s been on steroids for years. That’s because he eats a balanced, high-protein diet with generous…
Until her son, Will, was diagnosed with Duchenne muscular dystrophy as a 7-year-old, speech pathologist Tiffany Cook had never heard of the disease. Today, it’s literally her full-time job. As family resource manager with the nonprofit organization CureDuchenne, Cook — who works from her home in Dallas — helps…
The Christmas season has drifted in, and with it come mouth-watering kitchen smells. It’s time to mix the cookie dough, roll out the pie crust, and stick the turkey in the oven. I have fond memories of helping my mother make cranberry sauce from scratch. She was…
The University of Virginia (UVA) Children’s Hospital has been named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), making it the 22nd such center recognized by the patient advocacy group for the quality and comprehensiveness of its neuromuscular program. UVA’s hospital is also the first CDCC with a prominent…
Hoping to make travel less difficult throughout England for disabled motorists, including those with muscular dystrophy, Muscular Dystrophy UK and the government’s Department for Transport are working to bring accessible toilets to most highway service areas. Called Changing Places, the project is expected to be completed in the early…
For nearly half a year, Brenda Wong, MD, has led New England’s first and only interdisciplinary clinic devoted exclusively to boys and young men with Duchenne muscular dystrophy (DMD). The Duchenne Program at UMass Memorial Medical Center is located in Worcester, a 45-minute drive west of Boston. The clinic,…
The holidays can be a tumultuous time. Advertisements would have us believe that the ups and downs of life take a hiatus for the holiday season. However, many of us know that it’s unrealistic to expect that Christmas will be perfect. Our disabilities and chronic illnesses can be…
Early safety and efficacy data from a Phase 1 trial suggest that Taiho Pharmaceutical’s investigational compound TAS-205 may hold the potential to treat patients with Duchenne’s muscular dystrophy (DMD). The results were reported in the study, “A phase I…
Like many mothers of boys with muscular dystrophy, Deborah Hall hasn’t had an easy life. Hall, the daughter of a chemical engineer and a stay-at-home mom, was one of six siblings growing up in Boaz, West Virginia. When her younger brother, John, was 3 years old, doctors diagnosed him with…
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