Myotonic Dystrophy Foundation

SAN FRANCISCO (August 24, 2007) - The Myotonic Dystrophy Foundation (MDF) today formally announces public operations and its mission to lead and mobilize resources toward management, treatment and a cure for myotonic dystrophy (DM) through education, advocacy and research.

Myotonic dystrophy is the most common form of adult-onset muscular dystrophy affecting approximately thirty to forty thousand Americans and their families.  DM, the abbreviation of the Latin name dystrophia myotonica, is commonly misdiagnosed by the medical community.

The MDF’s primary near-term focus is to build and maintain a world-class Web site (www.myotonic.org) and to act as a clearing-house for information for DM-affected families, physicians and medical providers, as well as the research community. The MDF, a tax-exempt organization (Section 501 (c) (3)), will base its administrative and fiscal operations in San Francisco.  The MDF Board is currently comprised of seven  individuals whose families have been impacted by the disease. The MDF also collaborates closely with its Medical and Scientific Advisory Board, enlisted from the leaders of academic, clinical, and government centers. 

The MDF’s longer term goals are to advocate for and help fund research into treatment and eventual cure for DM. Towards this end, the MDF today also formally announces the creation of the MDF Excellence in Science awards, to be awarded bi-annually to promising young scientists and clinicians working in the DM field, and the MDF Award, to be awarded to an individual who has demonstrated sustained commitment to finding solutions for the disease. The awards will be presented at the International Myotonic Dystrophy Conference, to be held in Milan, Italy, in September (www.idmc-6.com). 

Author of the definitive work on myotonic dystrophy, Prof. Peter S. Harper of Wales, U.K., describes this degenerative, multi-systemic disease, as “…probably the most variable disorder found in medicine.”  Not only does it cause muscle atrophy and myotonia, but also complications in the cardiac, respiratory, endocrine, gastrointestinal, and skeletal systems and the brain, eyes, teeth, and hair. It can appear at any time from birth to old age.  It can cause low energy and motivation, excessive daytime sleeping, and problems with executive brain function. As a dominant genetic disorder, it can affect families vertically and horizontally, often presenting with different symptoms even among siblings. Since the gene was identified in the early 1990s, researchers have discovered that the genetic flaw generally enlarges and causes more severe symptoms in subsequent generations at an earlier age. This transmission of a more severe form at an earlier age, called anticipation, indeed renders myotonic dystrophy a “genetic time bomb.”

The MDF has formed strategic alliances with several independent organizations raising funds for DM research, including the Run America Foundation of Portland, Oregon; The Hunter Research Fund of Atlanta, Georgia; and the Stone Family Circle of Friends of Richmond, Virginia. The MDF expects to work with and complement the existing efforts of the Muscular Dystrophy Association (MDA), the National Institute of Health (NIH), the Centers for Disease Control (CDC), and other government and philanthropic agencies.

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If you would like more information about this new organization  please contact Lisa Vittek, Managing Director and Board Member of the Myotonic Dystrophy Foundation at lisa.vittek@myotonic.org.