(1) Spinal muscular atrophy (SMA) is the num-
ber one genetic killer of children under the age of 2. 
(2) SMA is an inherited and often fatal disease 
that destroys the nerves controlling voluntary muscle 
movement, which affects crawling, walking, head and 
neck control, and even swallowing. 
(3) It is estimated that SMA occurs in nearly 
1 of every 6,000 births and is therefore similar in 
incidence and severity to other well-known genetic 
diseases such as cystic fibrosis and Duchenne mus-
cular dystrophy, both of which may also benefit from 
additional focus and progress on SMA. 
(4) SMA is caused by the mutation of a single 
gene. This is extremely advantageous for genetic 
screening and therapeutic development. The gene 
mutation that causes SMA is carried by one in every 
40 people, or approximately 7,500,000 Americans. 
Each child of 2 carriers of the mutant gene has a 
1 in 4 chance of developing SMA. 
(5) The discovery of the gene responsible for 
the disease, SMN1, as well as a disease modifying 
‘‘back-up’’ SMN2 gene has opened the door to new 
SMA treatments. Modulating genes exist not only 
for SMA but also for other genetic disorders, includ-
ing Duchenne Muscular Dystrophy, Parkinson’s, and 
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Alzheimer’s disease. The modulation of these genes 
might be expected to impact these disorders. Success 
with SMN2 induction for SMA will serve as an im-
portant proof of principle and impetus for ongoing 
research in these other conditions. 
(6) Based on the advanced genetic under-
standing of SMA, the disease was selected by the 
National Institutes of Health (NIH) and the Na-
tional Institute of Neurological Disorders and Stroke 
(NINDS) as the prototype for the National Insti-
tutes of Health’s accelerated drug discovery effort, 
singling out SMA as the disease closest to treatment 
of more than 600 neurological disorders. 
(7) In 2003, the National Institute of Neuro-
logical Disorders and Stroke (NINDS) established 
the Spinal Muscular Atrophy Project: A Collabo-
rative Program to Accelerate Therapeutics Develop-
ment for SMA. The SMA Project’s unique collabo-
rative process between private, public, and non-profit 
partners provides a model translational research pro-
gram that can be replicated to accelerate the devel-
opment of safe and effective treatments for a wide 
variety of disorders. 
(8) National non-profit organizations dedicated 
to finding a treatment and cure for SMA continue 
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to provide substantial private funding and are col-
laborating with private biotechnology companies, 
large pharmaceutical companies, and clinical inves-
tigators to identify new drug compounds and facili-
tate the rapid translation of promising new therapies 
to individuals with SMA. The aforementioned invest-
ment by national non-profit organizations towards 
finding a treatment and cure for SMA is approxi-
mately equal, on an annual basis, to the resources 
committed by the Federal Government. 
(9) A Food and Drug Administration-approved 
SMA animal model exists that closely mimics the 
human disease. A number of therapeutics have been 
identified which are effective in animal models of 
spinal muscular atrophy. 
(10) There is an urgent need to provide Federal 
support enabling investigators to mount national 
clinical trials to demonstrate that these treatments 
are safe and effective for SMA patients. 
(11) The establishment and support of a na-
tional clinical trials network and a data coordinating 
center will promote rigorous patient evaluation using 
common protocols and allow investigators to study 
large numbers of patients to provide answers more 
rapidly than individual sites acting alone. 
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(12) There is a demonstrated need for greater 
interagency coordination on SMA research and in-
volvement by additional government partners to sup-
port the ongoing work of NINDS on the SMA 
Project as well the work of private SMA voluntary 
organizations, including most notably the need for 
active engagement by the National Institute of Child 
Health and Human Development (NICHD), along 
with support from the National Center for Research 
Resources, the Centers for Disease Control and Pre-
vention, the Food and Drug Administration, and the 
Health Resources and Services Administration 
(13) Despite such landmark legislation as the 
Orphan Drug Act and the Best Pharmaceuticals for 
Children Act, additional incentives for industry to 
engage early in the drug development process and 
through to drug approval are warranted for diseases 
as severe and devastating in infant and children 
populations as SMA. 
(14) Educating the public and health care com-
munity throughout the country about this dev-
astating disease is of paramount importance and is 
in every respect in the public interest and to the 
benefit of all communities. Furthermore, greater 
awareness of SMA may lead to the identification of 
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pre-symptomatic SMA-afflicted children, which has 
significant benefits relative to clinical trials and the 
search for a treatment and cure. 
SEC. 3. CLINICAL TRIALS NETWORK FOR SPINAL MUS-
CULAR ATROPHY. 
(a) CLINICAL TRIALS NETWORK.—The Director of 
NIH, in coordination with the Directors of the National 
Institute of Neurological Disorders and Stroke and the 
National Institute of Child Health and Human Develop-
ment, shall provide for the upgrading and unification of 
existing SMA clinical trial sites to establish a national 
clinical trials network for SMA. The Director of NIH shall 
ensure that such network— 
(1) conducts coordinated, multisite, clinical 
trials of pharmacological approaches to the treat-
ment of SMA; and 
(2) rapidly and efficiently disseminates sci-
entific findings to the field. 
(b) DATA COORDINATING CENTER.—The Director of 
NIH, in coordination with the Directors of the National 
Institute of Neurological Disorders and Stroke and the 
National Institute of Child Health and Human Develop-
ment, shall establish a data coordinating center with re-
spect to SMA to— 
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