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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.

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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.

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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.

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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?

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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.

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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.

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Research Briefs: BMD, DMD, EDMD, FA, LGMD, OPMD, Pompe disease, SMA

Santhera Pharmaceuticals announced May 9, 2011, that its drug Catena (generic name idebenone) appears to slow the decline in respiratory function associated with aging in people with Duchenne muscular dystrophy (DMD). Idebenone may improve energy production in muscle and nerve cells.The announcement, made at the 4th International Congress of Myology in Lille, France, reflects the first analysis of a two-year, open-label extension study (DELPHI Extension). (“Open-label” means everyone in the trial received Catena. There was no placebo group.)

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Enhancing Blood Flow to Exercising Muscles

Ronald Victor admits it: He never set out to study  muscular dystrophy. As an adult cardiologist specializing in hypertension (high blood pressure) and neurologic control of cardiovascular mechanisms, he’s a relative latecomer to the muscle field, but far from a reluctant one.Victor, who’s now associate director of the Cedars-Sinai Heart Institute and director of the Cedars-Sinai Hypertension Center in Los Angeles, has been interested for decades in how the body allocates blood supply to various tissues under different conditions — something that’s largely under the control of the autonomic nervous system.

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Bidets: A Disability Friendly Way to Go

A few years ago,  Bonnie Guzelf of Phoenix  began having difficulty using the bathroom by herself. Guzelf, who has a slowly progressive form of  ALS (amyotrophic lateral sclerosis), found that her hands, arms and legs were getting too weak to perform basic hygiene. A friend who is an occupational therapist suggested she get a bidet.

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Research Updates Spring 2011

In this issue:  MDA awards 44 new research grants * A recently discovered gene variant may indicate severity of DMD * Heart care in Duchenne MD and Becker MD addressed at MDA-sponsored meeting * Duchenne-Becker 'read-through' drug to begin development * Flow charts may aid in diagnosing CMT

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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 services are only available in the U.S.

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