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:
- 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.
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.
- ALS/Lou Gehrig's Disease
- Alzheimer's Disease
- Parkinson's Disease
- Deafness-Dystonia
- 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)
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:
- 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.
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.
- ALS/Lou Gehrig's Disease
- Alzheimer's Disease
- Parkinson's Disease
- Deafness-Dystonia
- 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)
We need to make a difference and we are negligent when there are things we can do and we do not do them.
This act is important to me because with the number of babies dying, we have to do something about it.
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3:15 pm PST, Jan 9,
Name not displayed, Illinois
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They need a chance to grow up.
This is important to me because it seems like a horrific way for a child to die...it makes me sick thinking of the pain they must suffer, and the suffer their parents go through watching as it happens, with no way to change the outcome. We rely on our scientific community to find cures because we are not scientists...I don't understand how 'they' can possibly say that they are cash strapped and cannot do anything about it. We are the richest nation in the world...there is no excuse.
I feel strongly about this petition. I'm a babysitter and I know a lot of children who would be miserable if they had this disease. It's just reminds me a lot about children with cancer. Just think about all of those kids who are in danger of their lives right now as we speak. Please, help out this petition and sign your name.
All children deserve a chance.
Any disability that can be addressed is of concern of all of us!