Recent Quest Articles
Why Does It Take So Long To Go from Mouse to Man?
John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported, where a treatment appears to cure one neuromuscular disease or another in a mouse — yet there are few treatments available today for people with any of these diseases, and only a few treatments in human clinical trials. Why does it take so long?Read More
Hit the Road in an Accessible RV
Barbara Graztke loves to vacation and enjoys traveling six to eight months out of every year. But she finds hotels uncomfortable and doesn’t like to fly. She visits family and friends across the country, but is unable to stay in their homes.The 65-year-old road warrior has post-polio syndrome and uses a power wheelchair. Barbara’s solution to making travel accessible is a 40-foot-long mobile home.Read More
MDA Funds Clinical Studies of Reformulated Drug for DMD
MDA has awarded $750,000 to Summit Corporation PLC for development and testing of SMT C1100, the company's experimental drug for treatment of Duchenne muscular dystrophy (DMD). Summit is an Oxford, United Kingdom, drug discovery company.The award was made through MDA's Venture Philanthropy (MVP) arm, a part of MDA's translational research program.Read More
Seeding the Field: Nurturing the Next Generation of MD Researchers
All MDA grants have the same basic goal: moving promising treatments through the “drug development pipeline,” from early discoveries in the lab, to clinical trials, to actual therapies that can be prescribed in the clinic.But some MDA grants are designed to accomplish this goal not only by funding research but by encouraging the “best and the brightest” young scientists to become future neuromuscular disease researchers.Read More
Front, Middle or Rear? Finding the Power Chair Drive System That's Right for You
Whether choosing your first power wheelchair or your next power wheelchair, picking the correct drive system for your needs is one of the first and most important decisions you’ll make.The location of the drive wheels (the wheels powered by the motor) in the rear, middle or front of the chair has a definite effect on the chair’s performance in different environments.Read More
Exon Skipping in DMD: What Is It and Whom Can It Help?
Exon skipping is a strategy currently being developed for Duchenne muscular dystrophy (although it may have application to other genetic diseases down the line) in which sections of genetic code are “skipped,” allowing the creation of partially functional dystrophin, the muscle protein missing in DMD.Read More
Going to the Emergency Room: Tips for People with Neuromuscular Diseases
When a medical emergency strikes — and the patient is a person with a neuromuscular disease — it’s not just getting to the emergency room quickly that’s critical. It’s also critical to ensure the ER staff understands the patient’s special needs caused by muscle disease.“Most ER doctors will NOT understand these diseases,” states Gregory Carter, medical director of the MDA Regional Neuromuscular Center at Providence St. Peter Medical Center in Olympia, Wash., and co-director of the MDA/ALS Center at the University of Washington Medical Center in Seattle.Read More
Declare Your Independence with a Wheelchair-Accessible Vehicle
Freedom. That’s what it’s all about, isn’t it? A wheelchair shouldn’t be a barrier to getting out and about, whether for work, day-to-day living or pleasure.Finding the right vehicle means analyzing your needs. Do you want to ride in your wheelchair or transfer to the vehicle’s seat? Will you be the driver or the passenger? If your muscle weakness is still progressing, how will your accessibility needs change down the line — and how can you accommodate them now?Read More
Research Briefs: The DMD/BMD-Affected Heart
In both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), deterioration of the heart muscle, a condition known as cardiomyopathy, is a major cause of disability and death.DMD and BMD both result from mutations in the gene for dystrophin, a protein found in muscles controlling body movement and respiratory effort, as well as in the heart. When dystrophin deficiency is complete (or nearly complete), the disease is known as DMD. When there's a partial deficiency of dystrophin, the result is BMD.Read More
FDA Approves Phase 1 Clinical Trial of RG3039 in SMA
In a historic first, biotech company Repligen Corp., of Waltham, Mass., has received approval from the U.S. Food and Drug Administration (FDA) to begin a phase 1 clinical trial of the experimental drug RG3039 for spinal muscular atrophy (SMA).This marks the first human trial of a drug specifically designed to treat SMA. RG3039 is a small-molecule compound designed to increase cellular levels of the SMN protein, which is deficient in SMA.Read More
MDA Resource Center: We’re Here For You
Our trained specialists are here to provide one-on-one support for every part of your journey. Send a message below or call us at 1-833-ASK-MDA1 (1-833-275-6321). If you live outside the U.S., we may be able to connect you to muscular dystrophy groups in your area, but MDA programs are only available in the U.S.