Individuals with spinal muscular atrophy (SMA) and their families recognized congressional champions and celebrated advocacy progress at Cure SMA’s Hope on the Hill event.
This year’s event, which was held on November 19 in Washington, DC, recognized several lawmakers, including U.S. Representative Sam Graves of Missouri, U.S. Representative Pete Stauber of Minnesota, and U.S. Representative Mark DeSaulnier of California, U.S. Senator Tammy Baldwin of Wisconsin, and U.S. Senator Shelley Moore Capito of West Virginia. Rep. Graves, Rep. Stauber, and Rep. DeSaulnier, who all attended in-person, spoke about their bipartisan efforts to make air travel safer and more accessible for passengers with disabilities who fly in a wheelchair through the passage of this summer’s aviation law. Sen. Baldwin and Sen. Capito spoke via video about their leadership work on the Labor-HHS-Education Appropriations Subcommittee in adding provisions to the FY 2024 and FY 2025 funding bills to support new SMA research at the National Institutes of Health (NIH). In addition, Sen. Baldwin highlighted her support of legislation to address the caregiving and transportation challenges faced by people with SMA and other disabilities. Cure SMA recognized other congressional leaders who have championed legislative priorities of the SMA community including the SSI Savings Penalty Elimination Act, Creating Hope Reauthorization Act, and the Safe Step Act.
The 2024 Hope on the Hill event also featured remarks from Dr. Joni Rutter, the Director of the NIH National Center for Advancing Translational Sciences (NCATS). Dr. Rutter spoke about NCATS’s work in bringing more treatments to all people more quickly through translational research, which advances research observations into health solutions such as treatments and therapies. Cure SMA President Kenneth Hobby provided an update at the event on the promising research pipeline for SMA. “We are on the cusp of multiple new and improved treatment breakthroughs in the year ahead, all of which are the result of decades of research collaboration between academia and government and industry with the support of the amazing SMA community.”
During the event, several SMA community members highlighted Cure SMA’s year-long advocacy efforts to help address the needs of children and adults with SMA.
Lyza Weisman, an adult with SMA from Colorado, spoke about SMA community advocacy to make air travel safer and more accessible for individuals who fly in wheelchairs. “Our collective education and Hill Day advocacy resulted in a new law that will get us closer to our accessible air travel goals.” The aviation bill was signed into law on May 16, 2024.
Vincent Pinti, an adult with SMA from Michigan, spoke about the U.S. caregiving crisis and its impact on the lives of individuals with disabilities. “Access to caregiving represents our independence, our community living, and our ability to achieve our individual dreams.” He highlighted legislation supported by Cure SMA to help individuals with SMA find caregivers and access the caregiving hours they need to maintain their health and independence.
Dave Gordon, a parent of a child who died from SMA as an infant in 1997, described his pride in the research and treatment success of the last decade. “Our past investments and research have changed the trajectory of the disease. But our work is not done. We’ll need the continued support and partnership of the NIH, FDA, researchers, and industry to help us realize our goal of finding a cure for the disease.”
Shannon and Greg Zerzan, who founded Hope on the Hill following their son’s SMA diagnosis, announced that the event had raised more than $2 million for SMA community research and support since the event began in 2011.
Cure SMA thanks CTIA, Genentech, and Scholar Rock for their lead sponsorship of the 2024 event.
