The U.S. Food and Drug Administration (FDA) in May approved edaravone (brand name Radicava) to treat ALS. Under development by Mitsubishi Tanabe Pharma America, Radicava is the first drug to be granted FDA approval to treat ALS in the United States in more than 20 years.Radicava is thought to work by relieving the effects of oxidative stress, which has been suspected to play a role in the death of nerve cells called motor neurons in people with ALS. (Oxidative stress is an imbalance between the production of free radicals and the ability of the body to counteract or detoxify their harmful effects with antioxidants.) Targeting this pathway could potentially preserve motor neuron health, which could in turn keep muscles functional for a longer period of time.
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Emergency situations present real challenges for individuals affected by neuromuscular diseases. For example, in February, when the Oroville Dam threatened to fail in California, downstream residents were given just one hour to leave their homes. Local news reported that a resident with a disability was left behind for hours due to lack of accessible transportation.
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At MDA, we believe our limits do not define us. Limits are meant to be overcome. Turning perceived limits into unlimited opportunities means different things to all of us. For some, living unlimited means taking on an extraordinary challenge, like summiting a mountain or skydiving. For others, it means making a new friend, going to college or pursuing a career goal. What these moments have in common is a sense of confidence and a willingness to live beyond what some may see as physical or societal limits created by living with a neuromuscular disease.
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Nine-year-old Lily Sander doesn’t remember life without Charcot-Marie-Tooth disease (CMT), but she has never let it hold her back. After undergoing major foot surgery three and a half years ago to correct the extreme curving of both feet caused by CMT, Lily had to learn how to walk again. She now walks with the help of leg braces and has been thriving.
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An upcoming Friedreich’s ataxia (FA) Patient-Focused Drug Development (PFDD) meeting with the U.S. Food and Drug Administration (FDA) is your opportunity to tell the FDA and drug developers about challenges and burdens you have experienced with FA, and share your thoughts about what is most important to you in evaluating potential new treatments for the disease.
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Dwight Koeberl, M.D., Ph.D., a pediatric medical genetics specialist at Duke University Medical Center in Durham, N.C., attended his first MDA Lock-Up six years ago as a guest. “I saw a patient at the clinic [whose family] was doing the MDA Lock-Up,” he says. “After I attended that with them, I have a sneaking suspicion they mentioned my name to MDA [as a potential Lock-Up participant], and that was how I got involved.”
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Some students with complex physical or cognitive needs have Individualized Education Plans (IEPs), and some have 504 plans (named for Section 504 of the Rehabilitation Act of 1973), while others may not elect to use these plans. Both plans are intended to assist students in gaining access to a free appropriate public education, but there are differences between the two methods. Here are some tips on how to choose a plan and implement it to improve your child’s educational experience.
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My understanding of the concept of independence recently blew up in my face. Part of independence, for me, has been my ability to live on my own. In fact, I live 1,200 miles away from any relatives. Sure, I cannot walk very far, but a massive part of embracing my freedom has been my ability to drive, to travel and to go where I want when I want.
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Elvira, a 16-year-old with spinal muscular atrophy (SMA), has been attending MDA Summer Camp near her hometown of Amarillo, Texas, since she was 7. Between campers, counselors and other volunteers, she has made a lot of friends and memories over the years, which is her favorite thing about attending camp.
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This is a busy and productive time for MDA and the families we serve. The unprecedented approval of three new drugs in six months to treat diseases in our program — after many years of investments with no new drugs — is proof that this is a time of great momentum in neuromuscular research. At MDA, we’re building on this progress as we push to do more to help families like yours in hometowns across America. As proof of our commitment to keep you at the heart of everything we do, here are some updates on new initiatives we’re developing for you and your family:
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