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Today is Rare Disease Day:  Protect NIH Research Funding from Sequestration by Contacting Your Congressman.

 February 28. 2013.

Patients and their families who feel isolated because of the rareness of their diseases should know that there are more than 6000 rare diseases affecting more than 60 million people in Europe and the US alone. Most of these diseases are genetic, serious, chronic and debilitating. Reaching out across borders can help them find common solutions and remind them they are not alone.  The 2013 theme is “Rare Disorders without Borders” Please see www.rarediseaseday.org for more information.

Importance of Rare Disease Day for SMA
While the focus of Rare Disease Day 2013 is “Rare Disorders without Borders”, this year marks several import events specific to the US.  2013 is the 30th anniversary of the Orphan Drug Act -- landmark legislation that encourages orphan drug development.  In 1983 only ten orphan drugs were approved by the FDA.  Today more than 400 have received FDA marketing approval.

Rare Disease Day as an important reminder about the need to address the unmet needs of people living with rare diseases.  Only a few hundred of the nearly 7000 rare diseases have approved FDA treatments.  Dedicated funding for earlier diagnosis, more basic research to understand disease mechanisms, as well as applied drug development will lead to faster treatments for rare diseases, including Spinal Muscular Atrophy.

"Sequestration” is scheduled to occur tomorrow.  These are across-the-board budget cuts for the federal government, including the National Institutes of Health (NIH) and Food and Drug Administration (FDA). These cuts have the potential for major impact on rare disease research and drug development funding.

Budget Cuts
The federal government’s investment in SMA research has never been more robust than at present. Thanks to families contacting their Members of Congress, resources and tools are being put in place at the NIH to support drug discovery efforts that will lead to a treatment for SMA.  Unfortunately, these efforts may be threatened if Congress permits deep spending cuts to take place.

Under current federal law, deep spending cuts are scheduled to go into effect automatically on March 1, 2013 unless Congress intervenes.  These cuts, known in budget terms as “sequestration,” will reduce the budget of several federal programs and threaten SMA research.  

The NIH is expected to be cut significantly in the sequestration process, which will be dramatically disruptive to the NIH’s medical research initiatives and significantly inhibit scientific discovery for years to come.  A critical bridge between Families of SMA’s seed efforts and the clinical trials supported by the biotechnology and pharmaceutical industry will be lost.  Several of the SMA-related research programs conducted or supported by the NIH are in jeopardy of being cut back, delayed, or terminated entirely under sequestration.  This will reverse a decade of progress in building greater support for SMA research within the NIH.

Contact Congress
Congress has the power to change the law and ensure that any deficit reduction efforts are carried out in a manner that does not threaten critical research for diseases such as SMA.  Contact your Senators and Representative to tell them to reverse sequestration and ensure that the NIH is not cut.

Talking Points 
I am calling to urge the [Congressman / Congresswoman / Senator] to amend the Budget Control Act and ensure that sequestration does not go into effect.
-Sequestration will cause the budget of the National Institutes of Health to be reduced, which would devastate research into lifesaving therapies for diseases such as Spinal Muscular Atrophy (SMA). 
-My [son / daughter / etc.] is affected by SMA, which is the leading genetic killer of children under the age of two.  The NIH is supporting several initiatives aimed at developing a treatment for SMA, many of which are very promising.  Sequestration will set-back this research.
-Please tell the [Congressman / Congresswoman / Senator] that I strongly urge [him / her] to protect the NIH from sequestration and to find a more equitable and fair way of reducing the deficit.

If you have any questions about NIH funding for SMA and the impact of sequestration, please contact Legislative Coordinator Spencer Perlman at spencer@fsma.org.


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