Gwendolyn DeBard Strong was born on October 4, 2007 and was diagnosed with Spinal Muscular Atrophy Type 1 (SMA1) in April 2008. SMA1 is a terminal genetic disease that results in loss of nerves in the spinal cord and weakness of the muscles connected with those nerves.
Her parents are asking that you consider signing a petition asking Congress to fund research into a cure for the disease. The NIH has said that a cure is possibly only a few years away.
The petition is here. Please read and sign it, and pass this on to others. The goal is 50,000 signatures. If each of you reading this sign now, we’ll get to that number in just a few hours. And if you watch the video, you’ll sign.
PLEASE SIGN THIS PETITION TO HELP CURE SPINAL MUSCULAR ATROPHY, THE #1 GENETIC KILLER OF CHILDREN UNDER THE AGE OF 2.
We need your help to move landmark legislation through Congress that will allocate federal resources to non-profit and research organizations focused on finding a treatment and/or cure for SMA.
o SMA is an inherited genetic disease that results in loss of nerves in the spinal cord and weakness of the muscles connected with those nerves.
o SMA is the #1 genetic killer of children under the age of 2.
o SMA is estimated to occur in nearly 1 out of every 6,000 births.
o The gene mutation that causes SMA is carried by 1 in every 40 people or nearly 7.5 million Americans.
o There is currently no cure, but the National Institutes of Health (NIH) and the National Institute of Neurological Disorders and Stroke (NINDS) have selected SMA as the disease closest to treatment of more than 600 neurological disorders.
o Researchers estimate that we are as close as only a few years away from finding a treatment and/or cure.
Our precious daughter, Gwendolyn (http://www.GwendolynStrong.com), was born perfectly healthy in October 2007 and diagnosed with SMA at the age of 6 months. SMA is a degenerative disease that destroys the nerves controlling voluntary muscle movement, which affects crawling, walking, breathing, head and neck control, and even swallowing. Gwendolyn has Type 1 SMA, which is the most aggressive, terminal form of the disease. Gwendolyn’s mind, heart, and spirit are no different from any other baby, but her body is failing her. We will most likely lose our little girl to this disease before she reaches the age of 2.
Gwendolyn is one of thousands of children coping with this devastating disease. In fact, 600 new babies will be born in the United States with SMA this year alone. The good news is hope is on the horizon. The National Institute of Health (NIH) and the National Institute of Neurological Disorders and Stroke (NINDS) have selected SMA as the disease closest to treatment of more than 600 neurological disorders and researchers estimate that we are as close as only a few years away from finding a treatment and/or cure for SMA. However, funding is needed to make that last and crucial leap. THAT’S WHERE WE NEED YOUR HELP!!!
For the first time, legislation has been proposed in the United States Congress to allocate federal resources to non-profit and research organizations focused solely on finding a treatment and/or cure for SMA. The SMA Treatment Acceleration Act (H.R. 3334/S. 2042) was introduced in the House of Representatives as H.R. 3334 by Rep. Patrick Kennedy (D-RI) and Rep. Eric Cantor (R-VA) and in the Senate as S. 2042 by Sen. Debbie Stabenow (D-MI) and Sen. Johnny Isakson (R-GA) in August 2007 and September 2007, respectively. This legislation is supported by Families of SMA, the SMA Foundation, Fight SMA, and the Muscular Dystrophy Association. The passage of this legislation could change the lives of thousands of children and give them the future they so deserve.
The SMA Treatment Acceleration Act specifically 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.
Although SMA has been selected by the NIH and NINDS as the closest disease to treatment of more than 600 neurological disorders and The SMA Treatment Acceleration Act will initially focus on SMA, the results and benefits will extend well beyond SMA. As researchers make progress unlocking a cure for SMA, their work is also making strides toward understanding and possibly curing a number of other rare and not so rare conditions. The following diseases and disorders will receive a “collateral benefit” from SMA research:
* ALS/Lou Gehrig’s Disease
* Alzheimer’s Disease
* Parkinson’s Disease
* Duchenne Muscular Dystrophy
* Fragile X, Friedreich’s ataxia
* Gaucher Disease
* GM2A (AB Variant of GM2 Gangliosidosis)
* Machado-Joseph Disease,
* Menkes Disease
* Metachromatic Leukodystrophy: Late Infantile
* Myotonic Dystrophy
* Neuronal Ceroid Lipofuscinosis (Batten Disease): Infantile, Late Infantile, Classic Late Infantile, and
* Niemann-Pick Disease (NPD)
* Sialidosis and Galactosialidosis
* Spinocerebellar Ataxia Type 1
* Spinocerebellar Ataxia Type 2/Episodic ataxia type 2
* Spinocerebellar ataxia type 6,
* Spinocerebellar Ataxia Type 7 (olivopontocerebellar atrophy with retinal degeneration)
* Tay-Sachs Sandhoff, and X-Linked Andrenoleukodystrophy (ALD)
As you know, legislation like this will only move through Congress with broad support and Members are significantly more likely to cosponsor and support legislation if their constituents are actively urging them to lobby for support of the bill on their behalf. Thus, to help move this legislation through the process WE NEED YOUR HELP IN SIGNING THIS PETITION to make sure your Senators and district Representatives know that this is an important piece of legislation to cosponsor.
As of July 12, 2008, there are 18 Senators and 63 Representatives in Congress cosponsoring this legislation.