Spinal Muscular Atrophy (SMA) is a hereditary disorder that destroys the nerves controlling voluntary muscle movement. It is the number one genetic killer of children under the age of two. SMA is a relatively common “rare disorder.” It is estimated that SMA occurs in about 1 in every 6,000 births. Approximately 1 in 40 individuals (7.5 million Americans) carry the gene that causes SMA, making it the second most common autosomal recessive genetic disorder. This incidence rate shows neither racial nor gender bias.

Presently, there is no known treatment for SMA, though there have been several exciting research breakthroughs over the past decade. Among more than 600 neurological disorders, SMA has been singled out by the National Institutes of Health as the disease closest to treatment based on scientists’ advanced genetic understanding of the disease.

In order to support the investigators, clinicians, and families who are working to find a treatment or cure for SMA, the SMA community, including Families of SMA, the SMA Foundation, Fight SMA, and the Muscular Dystrophy Association, has united behind the “SMA Treatment Acceleration Act.” This legislation authorizes federal funding in order 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 provides federal support to complement the substantial private funding that national non-profit organizations are investing to find a treatment for SMA. Passage of this landmark legislation will enable investigators to mount national clinical trials to demonstrate that identified therapeutics are safe and effective for SMA patients.

If you have any questions about the SMA Treatment Acceleration Act, or if you would like information about how you can become a cosponsor, please contact Spencer Perlman of Families of SMA (spencer@fsma.org / 202-333-5750), Laura Breiteneicher of the SMA Foundation (laurab@wswdc.com / 202-589-0800), Caroline Gibson of Fight SMA (carolinegibson@fightsma.com / 804-515-0080) or Annie Kennedy of the Muscular Dystrophy Association (akennedy@mdausa.org /202-828-8560).

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The year 2007 has been successful for the SMA community on Capitol Hill, most notably with the introduction of the SMA Treatment Acceleration Act in both the U.S. House of Representatives and U.S. Senate. This legislation (H.R. 3334/S. 2042) would provide increased federal support for spinal muscular atrophy (SMA) research and would strengthen a nationwide clinical trials network focused on approval of treatments for SMA. On behalf of our organizations and government relations teams, we are extremely grateful to the many individuals who have reached out to their Members of Congress to gain support for this important legislation. The list of cosponsors is growing each week! As it stands today, 30 Members of the House of Representatives and 10 Senators have signed on. Click here to see the current list. This is a very good early showing of support for this legislation and we are confident the ranks of supporters will grow even more in the New Year.

Again, our heartfelt thanks to each of you who have joined our efforts to advance the cause of SMA in Washington.

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The Savannah Morning News ran a great story yesterday about Skylar Swanson and her family of Richmond Hill, Georgia. After she and her twin sister Cassie were diagnosed with Spinal Muscular Atrophy ten years ago, Skylar’s family started SMA Angels Charity. Representatives of SMA Angels Charity, including the organization’s president and the twin’s grandmother Anne Meguiar and Board of Directors member Bubba Summerlin, have repeatedly joined FightSMA for its Annual Conference in Washington, D. C. and have met with Georgia’s Congressional representatives on issues including the SMA Treatment Acceleration Act.

From the article:

Butterflies follow 10-year-old Skylar Swanson.

They perch on her wheelchair, her suction machine and oxygen tube. Some even land on her strawberry blonde hair, on the tip of her finger or near her yorkie who rides on her lap.

The Richmond Hill fifth-grader, whose muscles are so weak she can’t walk or hold her head up, has a simple explanation for the butterflies.

Skylar says her twin sister, Cassie, sends them to her from heaven. Skylar’s identical twin has become her guardian angel.

When Cassie died last June, Skylar didn’t know a day without her twin.

The two girls were born with spinal muscular atrophy, an inherited neuromuscular disease that’s the No. 1 genetic killer of children younger than 2.

Up to one in 6,000 children are born with the disorder. Many are unable to stand, walk or even breathe without assistance.

The twins’ parents, David and Dana Swanson, had a choice: provide respiratory aid and ongoing, noninvasive care to keep the twins alive.

Or do nothing, beyond keeping the girls comfortable without breathing assistance, knowing the twins would die sooner.

They chose to fight for life.

To read the entire article, visit click here. Also, mark your calendars for SMA Angels Charity’s 10th So Many Angels Ball being held November 17th at the Savannah International Trade & Convention Center.

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On October 19th, the St. Petersburg Times ran an article about Trevor Smith and Laurie Elliot of the Tampa Bay chapter of FightSMA. Both Trevor and Laurie have urged Florida’s congressional representatives to support the SMA Treatment Acceleration Act.

From the article “Parents issue urgent plea to politicos”:

Thank God for the box of tissues. Trevor Smith couldn’t control her tears.

It could have been nerves. She was in Washington, D.C., without her husband or daughter for the first time.

Or maybe it was the gravity of what she had to tell C.W. Bill Young, the Bill Young, senior legislator known for getting the job done on Capitol Hill, face to face.

There was Trevor - 39-year-old wife of T.A. Smith, part-time sales rep for Kelly Services staffing agency and the mother of 6-year-old Joie - in a big, “stately looking” office.

She told Young about her daughter and about the disease that Trevor had never heard of until doctors diagnosed Joie. Spinal muscular atrophy, SMA for short, is the leading genetic killer of babies, according to the international nonprofit organization called Fight SMA.

On the Hill that day in April, dozens of regular people active in Fight SMA met with legislators or their aides. Laurie Elliott, whose 11-year-old daughter has the disease, had traveled from Hyde Park to sit across from Tampa’s own Rep. Kathy Castor.

They wanted to tell the people with political strength about an incurable disease that can lead to muscle weakness in children. They wanted to tell them that researchers think they can make a difference if they get more money for clinical trials and such.

To read the entire article, click here.

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We are very pleased to announce that legislation to enhance federal support for Spinal Muscular Atrophy (SMA) research, H.R. 3334, “The SMA Treatment Acceleration Act”, has been introduced in the U.S. House of Representatives by Congressman Patrick Kennedy (D-RI) and Congressman Eric Cantor (R-VA). U.S. Senator Debbie Stabenow (D-MI) and Senator Johnny Isakson (R-GA) plan to introduce companion legislation in the Senate when Congress returns from its August district work period. 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 and Senators Stabenow and Isakson for their leadership.

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 even breathing. Approximately one in 40 people, or approximately 7.5 million Americans, carry 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 the disease 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. Research involving the modulation of genes stands to benefit not only SMA, but other genetic disorders, including Duchenne Muscular Dystrophy, Parkinson’s, and Alzheimer’s. In recent Congressional testimony by the NIH, it was noted that “research on SMA illustrates the path from gene to understanding to treatment.”

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 FightSMA, Families of SMA, and the SMA Foundation, has united behind the SMA Treatment Acceleration Act, legislation aimed primarily at supporting a national clinical trials network for SMA.

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

• Federal support for a national clinical trials network for SMA;
• Federal support to enhance the existing SMA patient registry and for expanded research on the epidemiology of SMA;
• Establishes an SMA Coordinating Committee to include federal agencies, SMA researchers, and SMA families, which shall study barriers to development of SMA treatments;
• Establishes a trans-Institute research collaboration at NIH under the Director to ensure that all relevant Institutes are contributing and collaborating on SMA research;
• Requires the Secretary of the U.S. Department of Health and Human Services (HHS) to study and report to Congress on the use of incentives to promote SMA drug development among private industry;
• 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 will be issuing a “Nationwide Call to Action” for all SMA families, researchers, and friends, to help engage every Member of Congress in support of the this bill and the great efforts of Congressmen Kennedy and Cantor and Senators Stabenow and Isakson.

Cynthia Joyce
SMA Foundation

Kenneth Hobby
Families of SMA

Martha Slay
FightSMA

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

Laura Breiteneicher
SMA Foundation
laurab@wswdc.com

Spencer Perlman
Families of SMA
spencer@fsma.org

Caroline Gibson
Fight SMA
carolinegibson@fightsma.com

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We are very pleased to announce that Spinal Muscular Atrophy legislation, “The SMA Treatment Acceleration Act”, will be introduced in both the U.S. House of Representatives and the U.S. Senate in the next few weeks.We wanted to provide each of you, SMA families, researchers and friends, with a preview of this exciting step forward for all of our individual and collective efforts to raise awareness and secure more federal resources dedicated to finding treatments and a cure for SMA.

Once this legislation is introduced in the House and Senate, we also wanted you to know that we will be 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.

The proposed legislation, “The SMA Treatment Acceleration Act”, has been developed through a collaborative effort of the SMA Foundation, Families of SMA and FightSMA and is modeled on other disease-specific legislation currently before Congress or that has been previously considered and became law.

Specifically, “The SMA Treatment Acceleration Act” would provide for the following:

• Federal support for a national clinical trials network for SMA
• Federal support to enhance the existing SMA patient registry and for expanded research on the epidemiology of SMA
• Establishes an SMA Coordinating Committee to include federal agencies, SMA researchers, and SMA families
• Establishes a trans-Institute research collaboration at NIH under the Director of NIH to ensure all relevant Institutes at NIH are contributing and collaborating on SMA research
• Requires the Secretary of the U.S. Department of Health and Human Services (HHS) to study and report to Congress on ways to improve existing incentives, as well as to recommend additional incentives, necessary to promote SMA drug development among private industry
• The bill provides for the Secretary of HHS to establish a program to provide information and education on SMA to health professionals and the general public

The launch of this exciting initiative would not be possible without the active support of key Members of the House and Senate who have agreed to lead the effort as sponsors of this legislation. We look forward to joining with these Members in the official public announcement of the bill’s introduction, which we hope will occur no later than July. Some of these Members will be familiar to you as they have been long time friends of the SMA community and others are more recent and most welcome champions of SMA!

Lastly, let us again note the important role that we hope each of you can play to help ensure the success of this legislation. Getting a bill introduced in Congress is just the very first step and we will need your help to encourage each and every one of your Members and Senators to join the cause. It will take time, effort, patience, determination, and most importantly, your help!

Just as soon as the bill is formally introduced in the House and Senate, we will be issuing a “National Call to Action” on the SMA Treatment Acceleration Act where we will provide all the necessary materials for you to be able to call, write or email your Members of Congress to ask them to join the fight for treatments and a cure for SMA by cosponsoring the SMA Treatment Acceleration Act.

Sincerely,

Cynthia Joyce
SMA Foundation

Kenneth Hobby
Families of SMA

Martha Slay
Fight SMA

NOTE: If you have any questions, please feel free to contact any one of our Government Affairs staff:

Laura Breiteneicher, SMA Foundation: laurab@wswdc.com
Spencer Perlman, Families of SMA: spencer@fsma.org
Caroline Gibson, Fight SMA: carolinegibson@fightsma.com

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