Inclusion body myositis (IBM) is a rare, progressive muscle disease characterized by chronic muscle inflammation and weakness. It is estimated that approximately 20,000 people in the United States (US) have IBM, though the exact prevalence is unknown. IBM usually develops after age 50 and is more likely to affect men than women. The disease progresses . . .
The relationships we at MDA form with our community is one of the most rewarding parts of our mission. One such relationship is our friendship with Jon Burcaw and his son Shane. As a long-time supporter of Schaeffer’s Harley-Davidson – a member of the Eastern Harley-Davidson Dealership Association (EHDDA) – Jon became a supporter of . . .
For over 70 years, the Muscular Dystrophy Association has led the way as the #1 Voluntary Health Organization in the U.S. for people living with neuromuscular diseases. Through volunteer committees, boards, community events, and Summer Camps where kids build confidence and independence, volunteers are tightly woven into MDA’s fabric. We could not build on our . . .
On April 14, Edgewise Therapeutics announced the initiation of an Edgewise-funded observational trial in individuals with Becker muscular dystrophy (BMD). The goal of trial is to understand the progression of disease in individuals with BMD as assessed by functional measures and imaging endpoints. BMD is a genetic disease caused by a mutation in the dystrophin . . .
Name almost any sport, and it probably can be adapted for various abilities. That includes baseball, skiing, surfing, rock climbing, football, hockey, rowing, and more. The way a sport is adapted varies based on the type of equipment used. For example, there are wheelchair versions of tennis, curling, basketball, and rugby. Other sports use specialized . . .
Drug companies are developing more therapies for rare disease patients — and doing it faster — than ever before. But when it comes to enrolling minorities in clinical trials, the US track record is lagging behind. That subject was the focus of a March 15 session during the 2022 MDA Clinical & Scientific Conference in . . .
On April 5, an international research team, headed by Michela Guglieri at Newcastle University and Robert Griggs at the University of Rochester Medical Center, announced positive results at the American Academy of Neurology (AAN meeting) in Seattle, WA from the phase 3 FOR DMD study of corticosteroid dosing regimens for treatment of people with Duchenne . . .
Researchers at Goldsmiths University of London are seeking people living with muscular dystrophy (MD) and their primary caregivers to participate in a survey about their quality of life. The aim of the survey is to better understand how quality of life of an individual and their primary caregiver is affected by progressing MD. In this study, . . .
Researchers at Alexion Pharmaceuticals are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 2 clinical trial to evaluate the safety, effectiveness, and pharmacological properties of the investigational drug ALXN2050. ALXN2050 is designed to target a component of the immune system (known as complement), which underlies many autoimmune disorders including gMG. People . . .
Researchers at Alexion Pharmaceuticals are seeking adults living with dermatomyositis to participate in a phase 2 clinical trial to evaluate the safety, effectiveness, and pharmacological properties of the investigational drug ravulizumab (Ultomiris). Ravulizumab is designed to target a component of the immune system (known as complement), which underlies many autoimmune disorders including dermatomyositis. Treatment with ravulizumab . . .