Help Protect Federal Spending on Rare Diseases like Duchenne

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Help Protect Support of Federal Spending on Rare Diseases like Duchenne

Dear [Decision Maker],

As Congress debates the looming implementation of sequestration, we are writing as patients and families impacted by Duchenne Muscular Dystrophy to urge that you protect vital biomedical research, public health and medical innovation programs from these devastating across-the-board cuts.

A muscle wasting condition and the most common form of Muscular Dystrophy, Duchenne affects about one of every 3,500 boys, with about 20,000 new cases diagnosed worldwide each year. It is the most common lethal genetic childhood disorder and primarily affects boys because the Duchenne gene is found on the X chromosome. Patients today are generally wheelchair bound by their teenage years, and most patients live only until their late 20s.

Thankfully, as a direct result of biomedical and public health programs supported by Congress over the past decade hope is on the horizon.

Today about 20 potential Duchenne therapies and treatments are in various stages of the clinical trial pipeline, with additional potential therapies in earlier stages of development. Not too long ago, the Duchenne pipeline was virtually empty, but increased support for Duchenne research at the National Institutes of Health (NIH) has enabled significant advancements in science that have been translated into these potential treatments.

Also today, the life expectancy for the average patient with Duchenne is nearly 10 years longer than it was just a decade ago. This success was brought about largely by the efforts of the Centers for Disease Control and Prevention (CDC) to standardize care and better educate clinicians and family members about these care standards.

Under the terms of the sequester, funding for these vital biomedical research and public health programs would be drastically reduced, limiting our ability to achieve additional breakthroughs for those people affected by Duchenne and other serious and life-threatening diseases and conditions. In addition to biomedical research and public health initiatives, the terms of the sequester would reduce resources of the Food and Drug Administration (FDA) to thoroughly and expeditiously review new drug applications. The prospect of a situation whereby a promising potential therapy spends significantly more time undergoing review and evaluation because the agency lacks adequate staff resources is particularly disconcerting to the Duchenne community.

Advances in biomedical research, public health, and medical innovation have been responsible for increasing our lifespan, enhancing health, and producing a more prosperous nation.Thank you for your time.

[Your Name]
[Your Address]
[City, State ZIP]