Washington, D.C. – In an effort to combat the leading genetic cause of death in children under the age of two, U.S. Senator Sheldon Whitehouse (D-R.I.) has cosponsored legislation to accelerate research into spinal muscular atrophy (SMA), a disease that affects the Calise family of Cranston and thousands of others across the country. The Spinal Muscular Atrophy Treatment Acceleration Act (S. 1158) would rapidly advance treatments for SMA, a hereditary disorder that destroys nerves controlling voluntary muscle movement and occurs in nearly one in 6,000 births nationally.
“Researchers have already made great progress in studying potential treatments and cures for this deadly condition,” said Whitehouse. “I’m proud to join my colleagues, especially Rhode Island’s Patrick Kennedy, in an effort that will bring hope to thousands of children in our country.”
Whitehouse first met in 2007 with Michael Calise, whose daughter Corinna was diagnosed with spinal muscular atrophy at 11 months of age. Michael and Michelle Calise organized the Rhode Island chapter of FightSMA – Corinna’s Angels – in 2001. The Calises have advocated for additional funding for SMA in order to find better treatments and a cure for the genetic disease, which can affect crawling, walking, head and neck control and even swallowing. The mutated gene that causes SMA is carried by one in every 40 people; when two gene carriers have children, each child has a 1 in 4 chance of developing the potentially fatal disease.
“Simply put, we know what causes SMA, we know how to fix it, we just need the support and funding for extensive clinical trials to make it happen,” Michael and Michelle Calise said. “We realize that there are many worthy causes in this world. We are grateful that Senator Whitehouse has chosen to support SMA.”
According to the Spinal Muscular Atrophy Foundation, research over the past several years has advanced so remarkably that treatment – or a cure – for SMA may soon be within reach. The Spinal Muscular Atrophy Treatment Acceleration Act will direct the Secretary of Health and Human Services (HHS) to establish the Spinal Muscular Atrophy Coordination Committee to coordinate activities within the National Institutes of Health (NIH) and other federal health programs relating to SMA. Existing SMA clinical trial sites will be upgraded and unified into a national clinical trials network. The bill also directs the HHS Secretary, in consultation with the Food and Drug Administration (FDA), to report to Congress within six months and submit specific recommendations to improve and expand SMA drug treatment development.
The Spinal Muscular Atrophy Treatment Acceleration Act was authored by Senators Debbie Stabenow (D-Mich.) and Johnny Isakson (R-Ga.). Whitehouse is an original cosponsor.