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Recent Quest Articles
In Focus: Limb-Girdle Muscular Dystrophy
Say “limb-girdle muscular dystrophy” to most people, and you’ll get a questioning look. They may wonder what kind of girdle is being talked about and how limbs fit into the picture.The name actually comes from an anatomical term describing the supporting structures of the shoulders and hips, including bones and muscles. Limb-girdle muscular dystrophy (LGMD) is a genetic disorder in which the muscles surrounding the hips and shoulders degenerate.
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SMA: Full Speed Ahead
August is often referred to as Spinal Muscular Atrophy (SMA) Awareness Month, but for MDA, work on finding treatments for this degenerative disease — and providing assistance to individuals and families affected by it — is a year-round event.Today, research aimed at slowing, stopping or even preventing spinal muscular atrophy (SMA) is advancing with more speed and greater momentum than ever before. Here’s a roundup of some of the exciting research MDA has supported in the fight against SMA.
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CMD: Aiming Simultaneously at Two Biological Targets
Researchers at Boston University, supported in part by MDA, say their experimental two-pronged strategy for merosin-deficient congenital muscular dystrophy (MDC1A) was highly successful in a mouse model of this disease and should be further investigated as a potential treatment approach for patients.
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Taking Aim at Congenital Muscular Dystrophies
Update (March 24, 2015): A phase 1 trial of omigapil in children and adolescents 5-16 years old who have merosin-deficient congenital muscular dystrophy and meet other study criteria is open in Bethesda, Md. See Assessment of Safety and Tolerability of Omigapil (CALLISTO), or enter NCT01805024 in the search box at ClinicalTrials.gov.
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Service Monkeys Lend a Helping Hand
A guy rolls into his kitchen with a monkey on his shoulder … No, it's not the beginning of a really bad joke but an everyday occurrence for Michael Townsend of Baltimore, who received his monkey helper, Kathy, in 2007 from Helping Hands: Monkey Helpers for the Disabled. Townsend, 45, who has Duchenne muscular dystrophy and uses a power wheelchair, is one of more than 160 people across the country who has received a monkey helper free of charge from Helping Hands.
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All Aboard Accessible Amtrak
Eagerly anticipating the arrival of our train at the Hammond (La.) Amtrak station, my husband and I were as excited as the animated grade-school children running among the suitcases. All of us repeatedly gazed down the tracks trying to get the first glimpse of the engine light.Hammond is a 30-minute drive from our home and the second stop the “City of New Orleans” makes heading north to its final destination — Chicago. Months earlier, we had booked our reservation with Amtrak — after carefully comparing it to driving and flying.
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Living with MG: Personal Stories
Autoimmune myasthenia gravis is an unpredictable disease. On the one hand, it is a condition that can be effectively managed with medication and surgical interventions. On the other hand, even with treatment, symptoms may come and go, and treatments that once worked unaccountably may stop working.
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MG: Can the Immune Response Be Tamed?
It can start with nothing more than a drooping eyelid or a slight slurring of speech, either of which can come and go and improve with rest. But it can progress, often gradually over weeks or months, to affect all the voluntary muscles, including those controlling breathing. Weakness may be minimal early in the morning after a good night's sleep and worsen throughout the day, especially after periods of activity.
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Newborn Screening Recommended for Pompe Disease
The Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) today voted to add Pompe disease (acid maltase deficiency) to a list of diseases that it recommends states screen for in newborns.The list is known as the Recommended Uniform Screening Panel (RUSP).
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UPDATE: ACE-031 Clinical Trials in Duchenne MD
Update (May 2, 2013): In a May 2, 2013, press release, Acceleron Pharma and Shire PLC announced that they have concluded their collaboration on ACE-031 and related molecules, and will not restart the development of this program. John Knopf, CEO of Acceleron, said, "While we are disappointed in the outcome of the ACE-031 program, the research provided valuable insights that can be applied across Acceleron's pipeline of novel compounds and may benefit other patients in the future."
Read MoreMDA 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.
