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DMD Progression Studied in Very Young and Nonwalkers

Update (Jan. 23, 2013):  As of early 2013, MDA's DMD Clinical Research Network includes these five sites: University of California, Davis (UC Davis); Nemours Children's Hospital in Orlando, Fla.; Washington University in St. Louis; Nationwide Children’s Hospital in Columbus, Ohio; and Children's Medical Center in Dallas. See  Help Today, Help Tomorrow is Goal of MDA's Duchenne Clinical Research Network  to learn more.

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Viagra May be Heart Helper in DMD

Results from a recent study have shown that treatment with the drug sildenafil (brand name Viagra) conferred both long-term protection against cardiac (heart) dysfunction in younger mice, and rapid reversal of heart damage in aged mice with a disease resembling  Duchenne muscular dystrophy  (DMD).Cardiac disease in individuals with DMD or  Becker muscular dystrophy  (BMD) manifests most often as cardiomyopathy (heart-muscle weakness) or as an irregular heartbeat known as cardiac arrhythmia. The condition, often developing in childhood and the early teens in individuals with BMD or DMD, can result in congestive heart failure.

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New Apprenticeship Program for Personal Care Attendants

A new training opportunity, the Direct Support Professional Registered Apprenticeship Program, will assist direct support professionals (such as hired caregivers and aides) in advancing in this essential field, say the program’s creators, the National Alliance for Direct Support Professionals (NADSP) and the American Network of Community Options and Resources (ANCOR).

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Two-Drug Combo Makes a Difference in FA

Results from a clinical trial conducted in Spain involving 20 individuals with  Friedreich's ataxia (FA)  show that a combination therapy using two drugs, idebenone and deferiprone (DFP), demonstrated a "stabilizing effect" in neurological function and reduced abnormal heart-muscle enlargement in participants who received the two-drug combo for a period of 11 months.

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Low-Dose Ataluren Shows Some Benefit in DMD/BMD

A low-dose regimen of ataluren (formerly called PTC124), an experimental drug developed by  PTC Therapeutics  to treat  Duchenne muscular dystrophy (DMD)  or  Becker muscular dystrophy (BMD)  caused by a certain type of genetic mutation, is superior to a high-dose regimen or a placebo.PTC announced the findings at the 15th International Congress of the World Muscle Society, held in Kumamoto, Japan, Oct. 12-16, 2010, and in an Oct. 15 press release. (See  Pivotal Data Presented at the World Muscle Society Congress Suggest Ataluren Slows the Loss of Walking Ability in Patients with Nonsense Mutation Duchenne/Becker Muscular Dystrophy.)

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Immune Response Must Be Considered in DMD Gene Therapy

Editor's note: This story was updated April 27, 2012. Immune-system rejection of newly synthesized dystrophin protein occurred in at least some of the boys with  Duchenne muscular dystrophy (DMD)  who participated in a safety trial of dystrophin gene therapy.The finding was a partial surprise to researchers and demonstrates the value of small, phase 1 clinical trials.

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Cardiomyopathy in Becker MD

In many forms of muscular dystrophy, including  Duchenne muscular dystrophy (DMD)   and  Becker muscular dystrophy (BMD), weakness and degeneration of the cardiac muscle can be a major part of the disease, and one that frequently shortens life.Now, doctors at Ohio State University and Nationwide Children's Hospital in Columbus, Ohio, as well as Washington University in St. Louis and the University of Utah in Salt Lake City, have identified specific mutations in the dystrophin gene that predict whether heart muscle weakness (cardiomyopathy) will occur earlier or later in the course of BMD.

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A Dozen Ways to Ask for Help

I was taught it is better to give than to receive. Don’t ask for handouts. Pull your own weight.All my life, I told myself, “You are only handicapped if you allowed yourself to be handicapped.” That belief worked for me when I could still stand and walk. Now that my legs don’t function and my hands are extremely weak, I recognize that my physical being has limitations. I have readjusted my self-perception and understand that I need people to help me.  

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One Giggle at a Time

While cleaning a messy drawer, I began to smile and then chuckle at the foolish objects I have kept. When will I ever need a used twisty tie or a bent nail? And then there are the keys that don’t seem to fit anything. Am I just deceiving myself in believing these should be saved? Don’t laugh at me, laugh with me.

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James Lupski’s story has all the elements of a good sci-fi tale: a research scientist determined to uncover the cause of his own genetic disease, a friend and colleague who’s also an intrepid gene hunter, and a futuristic technology that not only pinpoints the molecular cause of the research scientist’s disease, but also opens the door to a new era of personalized medicine.

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