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Research without borders

Research without borders. Supporting research and patient outreach for FacioScapulo -Humeral Muscular Dystrophy (FSHD), the most prevalent form of muscular dystrophy.

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Research without borders

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  1. Research without borders Supporting research and patient outreach for FacioScapulo-Humeral Muscular Dystrophy (FSHD), the most prevalent form of muscular dystrophy. Progress on FSHD could benefit a wide range of other areas of medicine, from cancer and diabetes to muscle regeneration and repair.

  2. What is FSHD? • FSH Muscular Dystrophy (FSHD) is caused by a genetic defect, one that is passed down from parent to child but can also arise spontaneously. It can affect anyone. • An estimated 500,000 people worldwide have FSHD, and 1-2% of the population carries a risk gene. • The disease attacks all skeletal muscles – typically affecting the face, shoulders, arms, back and legs. • Symptoms may be evident at birth or during childhood, but more often appear during teenage and adult years. • FSHD can be profoundly disabling, causing a loss of facial expression, difficulties with speech and hearing, and an inability to lift objects or walk.

  3. What the FSH Society has Accomplished • Created a global research program when none existed, by recruiting and funding scientists to undertake FSHD research. • Persuaded the U.S. government to allocate funding to FSHD research. • Convened international research meetings to coordinate a global campaign to cure FSHD. • Funded work leading to the discovery of FSHD genes and paving the way to potential treatments. • Empowered a worldwide network of patients and families through education, peer-to-peer support and access to care.

  4. Scientific Breakthroughs Made Possible by FSH Society Funding • The discovery of a specific genetic “package” called DUX4 that is associated with most cases of FSHD. • The successful reversal of FSHD symptoms in mice using gene therapy (RNAi). • The identification of genes and proteins that damage muscle cells, as well as the mechanisms that can cause the disease. • Developed FSHD cell lines and animal models to test hypotheses and potential treatments.

  5. Why Your Support is Critical • With enough funding, we could have the first effective FSHD treatments in the next 5-10 years. • NIH budget cuts are slowing down progress. Top scientists spend more time writing grants instead of working in the lab. • Without enough grants, some may have to stop their research, jeopardizing years of public and private investment in their work. • We cannot afford to slow down. Patients need treatments in their lifetimes! • FSHD research may help in other areas of medicine, from cancer to diabetes as well as other muscle-wasting disease that are more common.

  6. Give with Confidence FSH Canada Foundation partners with the FSH Society to direct funding to the world’s best research, guided by the Society’s world-renowned Scientific Advisory Board. The FSH Society has earned its fourth consecutive 4-star Charity Navigator rating, awarded to charities that outperform the majority of organizations. Eighty-seven percent of the revenue raised by the Society goes directly to research and education.

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