NIH Conference and Legislative Update

Dear Families,

Thank you for all you have done to increase awareness and attention towards Spinal Muscular Atrophy (SMA). As most of you are aware, this has been an eventful year in the U.S. Congress; national health care and research priorities have been prominent on the radar screens of lawmakers. The significant visibility that you have brought to SMA through outreach to policymakers has yielded significant research and policy momentum for our community. Since it has been several months since we last provided a joint update on such activities, we wanted to take a moment to inform you about recent and upcoming government-sponsored, SMA-related research conferences and to provide an update on the SMA Treatment Acceleration Act (H.R. 2149 / S. 1158).

This fall, several landmark federal research meetings are being conducted to address critical regulatory and therapeutic development opportunities in SMA. In September, the Food and Drug Administration (FDA) and the National Institutes of Health (NIH) jointly hosted a research conference focused on Antisense Oligonucleotide Therapies. If you were unable to view the live stream of this meeting, a summary will soon be available from NIH. Additionally, this week, the NIH is sponsoring a Therapy Development Workshop. For information about viewing the live stream of this week’s NIH SMA Workshop, please go to http://www.sonicfoundry.com/NIH.

With regard to the SMA Treatment Acceleration Act, despite our best efforts and the unbelievable show of support by families across the nation, the current political climate in Washington makes it highly unlikely that the bill will pass this year.

After extensive conversations over the past several weeks with senior staff persons and key Members of Congress who serve on the congressional committees with jurisdiction over the bill, it has become clear that very few, if any, public health bills such as the SMA Treatment Acceleration Act will move through Congress during the lame-duck session set to begin on November 15. Furthermore, those bills that may move have been revised considerably and we believe that weakening the SMA Treatment Acceleration Act is unacceptable to our community and is too high a price to pay in order to achieve a symbolic gain.

While we are all disappointed by this realization, we want you to know the following:

1.  Even if the bill, as expected, does not pass in the lame-duck session, the overall effort to push the legislation forward has had an incredibly positive impact; it has dramatically raised awareness of SMA among lawmakers and is directly responsible for the increasing attention SMA is receiving from the National Institutes of Health (NIH).

2.  We will work with our champions on Capitol Hill to evaluate and introduce a new version of the Act in the next (112th) Congress, while continuing to stay in close contact with key Members and their health staff in case circumstances change before the end of this year. Looking ahead, we will review the clinical research landscape and work with legislators to craft legislation for the 112th Congress that addresses the specific needs of the research community.

While it is unlikely that the SMA Treatment Acceleration Act of 2009 will pass in the coming weeks, we believe that this is a time to celebrate the remarkable efforts of the entire SMA community in driving forward the Federal Government’s interest in SMA research. The bill has had a tremendously positive impact to date. Your efforts in securing 118 cosponsors in the House and 21 cosponsors in the Senate are responsible for the opportunities that are emerging to engage the NIH even more deeply in the effort to find a treatment for this disease.

We continue to be eternally grateful to you for your efforts and hope that you will remain energetic, engaged, and hopeful as we continue in our legislative and governmental efforts. Thank you for all that you do. We would not have achieved so much without your personal contributions and outreach to Congress and we have much to be proud of.

THANK YOU AGAIN for your help for all those with SMA!

Sincerely,