FightSMA representatives Martha Slay and Caroline Gibson traveled to Washington, D.C. earlier this week, to garner support and cosponsors for the SMA Treatment Acceleration Act (HR 2149, S 1158).

As the healthcare debate continues in Congress, we are working hard to keep our message and our mission front and center on Capitol Hill: the passage of the first-ever federal legislation dedicated to spinal muscular atrophy.

Because the Energy & Commerce committee is the committee of jurisdiction in the House, FightSMA visited Committee Members offices, sharing information on the bill with Health Staff, as well as signatures from PetitionToCureSMA.com.

Offices Visited
Rep. Mike Ross (AR)
Rep. John Barrow (GA)
Rep. Mike Doyle (PA)
Rep. Charlie Melancon (LA)
Rep. Tim Murphy (PA)
Rep. Cliff Stearns (FL)
Rep. Edward Markey (MA)
Rep. Eliot Engel (NY)
Rep. John Dingell (MI)
Rep. Zach Space (OH)
Rep. Baron Hill (IN)
Rep. Bart Stupak (MI)
Rep. Nathan Deal (GA)

In addition to House Energy & Commerce Committee Member offices, FightSMA also visited in Congressman Patrick Kennedy and Congressman Eric Cantor’s offices, our lead House sponsors, to thank them for their hard work and dedication to finding a cure for SMA.

Click here for more information on the SMA Treatment Acceleration Act.

Click here to sign the Petition To Cure SMA.

To contact your Members of Congress, please contact CarolineGibson@FightSMA.com

Congressman Eric Cantor (R-VA) meets Greg Tapley from FightSMA’s Georgia chapter, following the recent FightSMA Conference in Washington D.C. Greg thanked Congressman Cantor for his sponsorship of the SMA Treatment Acceleration Act, which he and Congressman Patrick Kennedy (D-RI) re-introduced in the House of Representatives on April 28, 2009.

We are very pleased to announce that legislation to enhance federal support for Spinal Muscular Atrophy (SMA) research, H.R. 2149, the “SMA Treatment Acceleration Act of 2009“, has been introduced in the U.S. House of Representatives by Congressman Patrick Kennedy (D-RI) and Congressman Eric Cantor (R-VA).  On behalf of our organizations and the families affected by this deadly disease, we want to express our gratitude and thanks to Congressmen Kennedy and Cantor for their leadership.

This legislation builds upon the success of the SMA Treatment Acceleration Act introduced in the 110th Congress, which garnered 85 cosponsors in the House and 21 in the Senate, including then-Senator Barack Obama.  The new version of the legislation has been modified slightly to ensure resources for SMA clinical efforts and infrastructure are maximized.

SMA is the number one genetic killer of children under the age of two.  It is an inherited disease that destroys the nerves controlling muscle movement, which affects crawling, walking, head and neck control, swallowing, and breathing.  Approximately one in 40 people, or approximately 7.5 million Americans, carries the gene mutation that causes SMA.  Each child of two carriers of the mutant gene has a one in four chance of being afflicted by SMA.

Among more than 600 neurological disorders, SMA has been singled out by the National Institutes of Health (NIH) as one of the diseases closest to treatment based on scientists’ advanced genetic understanding of the disease and a strong collaboration between families, federal agencies, and patient advocacy groups.  Researchers have identified the gene responsible for SMA, as well as a disease modifying “back-up” gene that has opened the door to promising new treatment pathways.  This research is providing groundbreaking data for SMA and other disorders, including the muscular dystrophies, Lou Gehrig’s disease, Friedriech’s Ataxia, Fragile X syndrome, and Huntington’s disease.

In order to build on the progress being made by investigators and bring treatments to children affected by SMA, a broad coalition of organizations, including Families of SMA, FightSMA, the Muscular Dystrophy Association (MDA) and the SMA Foundation, has united behind the SMA Treatment Acceleration Act of 2009, legislation aimed primarily at supporting a national clinical trials network for SMA.

Specifically, the “SMA Treatment Acceleration Act of 2009” provides for the following:

• Federal support for a national clinical trials network for SMA;
• Federal support to enhance the SMA patient registry and for expanded research on the epidemiology of SMA;
• Establishes an Interagency SMA Research Coordinating Committee to include federal agencies including NIH, SMA researchers, and SMA families, to coordinate government activities relating to SMA, develop a comprehensive strategy for improving and expanding SMA research, make recommendations to strengthen collaborative research across multiple institutes at NIH, and identify barriers to the development of drugs for treating SMA; and
• Provides for the Secretary of HHS to establish a program to provide information and education on SMA to health professionals and the general public

Our organizations are issuing a “Nationwide Call to Action” for all SMA families, researchers, and friends, to help engage every Member of Congress in support of this bill and the great efforts of Congressmen Kennedy and Cantor.

Kenneth Hobby
Families of SMA

Martha Slay
FightSMA

Annie Kennedy
MDA

Cynthia Joyce
SMA Foundation

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NOTE: For more information on the “The SMA Treatment Acceleration Act” please contact any one of our Government Affairs staff:

Spencer Perlman, Families of SMA, spencer@fsma.org

Caroline Gibson, FightSMA, carolinegibson@fightsma.com

Annie Kennedy, MDA, akennedy@mdausa.org

Laura Lay, SMA Foundation, llay@wswdc.com

Click here to download the Joint Announcement.

RICHMOND, June 6, 2008 — This week, the entire Virginia delegation to Congress has unanimously signed on as co-sponsors of the SMA Treatment Acceleration Act, a bill designed to increase federal support for research to develop a treatment for spinal muscular atrophy (SMA), the number-one inherited cause of infant death.

“This tremendous show of support and leadership will bring hope to thousands of families battling this deadly neuromuscular disorder,” said Martha Slay, president of Richmond-based FightSMA, one of the groups that is supporting the legislation.

“The Virginia delegation is setting a wonderful, bipartisan precedent for the rest of Congress and the Senate where we need to continue to build support this year for this legislation,” said Ms. Slay. “It is immensely gratifying that our home state has come through in such a big way.

“We particularly appreciate the support of Congressman Eric Cantor of Richmond, who was the original sponsor, along with Congressman Patrick Kennedy of Rhode Island. We would not be where we are today without Congressman Cantor taking the lead on this legislation,” said Ms. Slay.

The SMA Treatment Acceleration Act authorizes federal funding to:

• Upgrade and unify existing SMA clinical trials sites and establish a national clinical trials network for SMA.
• Establish a Data Coordinating Center to provide expert assistance and advice to SMA clinical trials sites.
• Expand and intensify federally supported research programs with respect to pre-clinical translational research related to SMA.
• Establish a research collaborative at the National Institutes of Health to ensure cooperation across multiple Institutes regarding research related to SMA.
• Enhance and provide ongoing support to the existing SMA patient registry in order to provide for expanded research on the epidemiology of SMA.
• Establish an SMA Coordinating Committee, consisting of representatives from relevant government agencies and the public, to coordinate government activities relating to SMA, serve as the principal advisor to agency heads, and conduct a study to identify barriers to the development of drugs for treating SMA and report findings and legislative recommendations to Congress.
• Require the Secretary of Health and Human Services to collaborate with the FDA and the Coordinating Committee to make recommendations for improving and expanding existing industry incentives to promote SMA drug development.
• Establish and implement a program for providing information and education on SMA to health professionals and the general public related to advances in the diagnosis and treatment of SMA and the provision of care to SMA patients.

The SMA Treatment Acceleration Act is supported by Families of SMA, FightSMA, the Muscular Dystrophy Association, and the SMA Foundation.

Co-sponsors from Virginia are: Senator John Warner, Senator Jim Webb, Congressman Eric Cantor, Congressman Rick Boucher, Congressman Jim Moran, Congresswoman Thelma Drake, Congressman Robert Wittman, Congressman Bobby Scott, Congressman Randy Forbes, Congressman Virgil Goode, Congressman Bob Goodlatte, Congressman Frank Wolf, and Congressman Tom Davis.

FightSMA, a nonprofit 501c3 corporation, was founded in Richmond, VA, in 1991 with the mission “to strategically accelerate research for a treatment and a cure for spinal muscular atrophy (SMA), the leading genetic cause of infant death.”

For more information, contact FightSMA at www.fightsma.org or 804.515.0080.

At the recent 2007 FightSMA National Spinal Muscular Atrophy Conference, Congressman Patrick Kennedy of Rhode Island addressed the attendees about spinal muscular atrophy research funding and the SMA Treatment Acceleration Act. The proposed legislation, developed through a collaborative effort between FightSMA, the SMA Foundation, and Families of SMA, would establish a national clinical trials network to develop treatments that are safe and effective for SMA patients.

Conference attendee Karin Vallo captured the speech on video. Karin is a leader in the Desiree’s Buddies/FightSMA Southwest Chapter in Albuquerque, New Mexico, and is the grandmother of Desiree. A big “thank you” goes out to Karin for helping us make this video available!

Sometimes it’s hard to see turning points until you’re past them. We here at Fight SMA hope that we’re at one now. Spinal muscular atrophy is the leading genetic killer of children under two, and currently there’s no cure. However, the National Institute of Neurological Disease and Stroke (NINDS) of the National Institutes of Health (NIH) says that out of the 600 disorders it tracks, SMA is one of the closest to being cured. This is based on advanced genetic understanding of the disease by scientists. This is not only good news for the SMA fight. Research into spinal muscular atrophy benefits more than 40 other genetic and neurological disorders, including ALS (Lou Gehrig’s disease), Parkinson’s, Alzheimer’s, and Duchenne Muscular Dystrophy.

Meanwhile, Congressman Eric Cantor of Virginia, a long-time friend of Fight SMA and the SMA Community, is sponsoring the SMA Treatment Acceleration Act. The proposed bill is a collaborative work of the SMA Community, led by Families of SMA, Fight SMA and the SMA Foundation. It would establish a national clinical trials network to develop treatments that are safe and effective for SMA patients. Congressman Cantor is asking that his colleagues in the House and Senate join him in sponsoring this bill.

At the annual Fight SMA Spinal Muscular Atrophy Conference in Washington D.C. last week, The SMA Day on the Hill was highly successful. Our friends and families visited more than 65 congressional offices, meeting with Congressmen and women and their staff to advocate on behalf of spinal muscular atrophy. The effort was effective in enlisting the support of a number of legislators on both sides of the aisle.

The perfect close to the day of visits on Capitol Hill was a reception hosted by Congressman Patrick Kennedy (D-RI) in the Rayburn House Office Building. Congressman Kennedy (shown at the left meeting conference attendees Malorie Fox and Erinne Williams - click the image for a larger version) is advocating within the House Appropriations Committee for SMA support. He gave a powerful speech to our group, which we hope to post on this blog as soon as the video is prepared. He also visited with all the families and friends after he spoke and took time out to take pictures with all of them.

Fight SMA joins with the entire SMA Community in deep appreciation of the advocacy and efforts by Congressman Cantor and Congressman Kennedy. We also urge all of our nation’s lawmakers to recognize the opportunity of The SMA Treatment Acceleration Act to open doors for SMA and countless other diseases.

We have much to celebrate right now. There is still much work to be done, but we feel a victory is absolutely forthcoming.