On Tuesday, Cure SMA staff members and family advocates, and clinicians from the University of Utah, met with Senator Orrin Hatch (R-UT) to discuss newborn screening for spinal muscular atrophy. Senator Hatch was an original sponsor of the Newborn Screening Saves Lives Act of 2007, which established federal programs to support states in the implementation of newborn screening, provided resources for education to parents and healthcare providers, and helped ensure state labs met quality standards for screening.
This law also includes the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children. This committee reviews conditions for the Recommended Uniform Screening Panel (RUSP). SMA is currently being reviewed for inclusion on the RUSP, with a decision anticipated in February 2018.
Family advocates also discussed with Senator Hatch the importance of continued funding for NIH research and legislation like the Newborn Screening Saves Lives Act and the Orphan Drug Act, which helps facilitate development of orphan drugs — drugs for rare diseases including SMA.
Later in the day Cure SMA hosted a luncheon for state legislators to educate them on the science of treating SMA and to discuss state implementation of and funding for newborn screening.
Utah will begin screening for SMA on a pilot basis in 2018. Pilot screening projects are often a prelude to the implementation of permanent screening for a new condition. States use the learnings from these pilots to ensure efficient and effective implementation.
In addition to Utah, several other states, including Wisconsin, Massachusetts, North Carolina, Georgia, and Minnesota plan to begin pilot screening in 2018. The pilot programs in Utah and North Carolina are funded by Cure SMA.
These states will join New York, which is already screening on a pilot basis, and Missouri, which will implement screening in 2018 after the legislature passed a first-in-the-nation bill adding SMA to their state’s permanent screening panel.
Thank you to the University of Utah clinicians and family advocates that made this day a huge success!
Thank you to Dr. Nicholas Johnson, Assistant Professor of Neurology, Pediatrics, and Pathology, University of Utah; Dr. Russell J. Butterfield, MD, PhD, Clinical Neurosciences Center, University of Utah; Stephanie Bradshaw, family advocate; Serei Panh, family advocate; Travis, Holli and Brooklyn Hensley, family advocates; and Elliot, Janell, Evie and Angel Blakely Lewis, family advocates.