When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful or no different than available alternatives. Investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants. Phase 0 — Also known as “exploratory studies,” these trials test a small dose of a new drug in a few people as researchers explore how the drug may work in humans and ensure that it is safe. Trial participants may or may not have the disease the drug has been developed to treat.
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Keeping pace with the latest assistive mobility products is a tall order. The marketplace is so dynamic, so innovative, so accelerated; it can be impossible to stay on top of what’s new, what’s different, what’s covered by insurance or what will enhance your mobility and independence. This edition of “Innovation” catches up with some of the smartest assistive mobility products on the market now, as well as tips for selecting the right types to meet your needs.
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Between working as a resident assistant, being the philanthropy coordinator for her sorority Alpha Gamma Delta, acting as the 2016 Greek Sing Director, planning a study abroad trip to Australia and volunteering for other organizations, it’s hard to believe that Sarah Bellish, a sophomore at Baldwin Wallace University (BWU) who has spinal muscular atrophy (SMA), still has the time to advocate for improved accessibility on her college campus.
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Ava Illingworth picked up running two years ago as an outlet to burn off some of the stress from her job installing software and interfaces for health care organizations. It wasn’t the most likely stress reducer for her, considering she has been living with Charcot-Marie-Tooth disease since she received the diagnosis in 2009 at the age of 35.
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Sports have always been a big part of Jermia White’s life. White, a 23-year-old sports statistician from Dallas who has spinal muscular atrophy (SMA), remembers watching her father coach basketball when she was 9 years old, and she went on to be a cheerleader, a manager for her high school basketball team and sports editor for her school’s newspaper. Before she went off to college, she knew she wanted to be involved with sports, but it wasn’t until she got to Dallas Baptist University that she figured out the right fit.
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Members of the MDA community across the country have come together to walk a staggering amount in the name of neuromuscular disease research this year. In 2016 alone, there were:• 145 Muscle Walk finish lines crossed• More than 280 miles of Muscle Walk routes• 48 participating states• 363 hours of Muscle Walk events
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Joe Feidt’s 40-year love affair with the sport of disc golf began as a happy accident. Feidt, a 66-year-old writer and editor for DiscGolfer magazine who has inclusion-body myositis (IBM), first discovered the sport in 1976 at a Frisbee tournament in Minneapolis. As it happened, one of his best friends from college was already on the pro Frisbee tour, and he encouraged Feidt to get involved.
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When Tia Blankenship was diagnosed with Charcot-Marie-Tooth disease (CMT) in 2002 at the age of 23, she was told she wouldn’t be able to walk by the time she turned 35. This past April, at the age of 37, Blankenship walked both the 5K walk and the 1-mile walk at the MDA Muscle Walk of Tampa Bay (Fla.).
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An important win for all individuals and families living with muscular dystrophy, ALS or one of the related neuromuscular diseases that MDA fights occurred Sept. 19, 2016.The U.S. Food & Drug Administration (FDA) granted accelerated approval to eteplirsen (brand name Exondys 51) for the treatment of some forms of Duchenne muscular dystrophy. This is an outcome MDA dreamed of decades ago when we first invested in the research to identify the cause of DMD and then took the risk to be the first to invest in a breakthrough exon skipping research project that led to development of this drug (see Progress Now). It is the first of what we know will be many disease-modifying drugs for DMD.
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Are you excited about the progress we are making in bringing strength, independence and life to individuals with neuromuscular diseases and their families?This holiday season, please remember to put MDA on your list. Please join us in supporting the important work being done to find research breakthroughs across diseases that accelerate treatments and cures; care for kids and adults from day one at more than 150 MDA Care Centers; and empower families with services and support, including equipment assistance, support groups and MDA Summer Camp for kids.
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