On the cover: Andy Vladimir, of Coconut Grove, Fla., has MMD and uses a wheelchair. This hardly slows him down. A travel writer and textbook author, he’s a frequent contributor to MDA Quest magazine’s “To Boldly Go” column. Dear Friends What is Myotonic Muscular Dystrophy? What causes myotonic muscular dystrophy? What happens in adult-onset MMD? Comparison of MMD1 and MMD2 What happens in congenital MMD? How is MMD diagnosed? How is MMD treated? Does it Run in the Family? MDA's Search for Treatments and Cures MDA is Here to Help You

MDA’s Search for Treatments & Cures

The years since the discovery of the genetic cause of MMD in 1992 have been fruitful ones for MMD research.

Scientists, many of them funded through MDA’s worldwide research program, are gaining understanding of how the expanded DNA sections on chromosome 19 and chromosome 3 cause so many physiologic changes. Such discoveries are likely to provide valuable insights for future treatment avenues.

In the meantime, a compound based on a body protein called IGF1, which experiments have shown may promote muscle growth, is undergoing testing.

The ultimate “cure” for MMD1 and MMD2 probably will require finding a way to block the expanded area of DNA on chromosome 19 or chromosome 3 so that it loses its toxic effect on cells. It’s no longer far-fetched to imagine that in the future, this expanded section of DNA could be blocked or “silenced.” MDA grants are supporting development of this type of treatment.

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