On November 30, members of the SMA community met with 17 different legislative offices— eight from the Senate and nine from the House of Representatives. 

These Senators and Representatives serve on committees important to our community’s goals, including the House Energy & Commerce Committee; Senate Health, Education, Labor and Pensions (HELP) Committee; the House and Senate Appropriations Committees; and the Labor-Health and Human Services-Education Appropriations Subcommittees. Many also are members of the Rare Disease Caucus in Congress. 

The members of the community conducting these visits included families impacted by SMA, Cure SMA staff, and other partners from our community. 

The staff and legislators expressed appreciation to the SMA community, particularly their gratitude for the families who shared their stories to help educate Congress on the impact of SMA. They also asked to be kept apprised of developments, including the planned application for SMA to the newborn Recommended Uniform Screening Panel (RUSP), and the current New Drug Application (NDA) pending with the FDA. 

Follow-up visits are being planned for early 2017. 

Building Relationships with Government and Regulators

These Congressional meetings were part of the activities surrounding the 6th Annual “Hope on the Hill” Congressional Dinner, a gathering of more than 160 people from the SMA community— including government and industry partners. 

As more SMA drugs reach late stage clinical trials and progress toward FDA approval, we remain focused on educating, involving and engaging all three groups in our community: the patient and family group, the government and regulatory group, and the biotech and pharmaceutical companies that are developing and testing SMA drugs. We recognize that we need the whole community working together in order to develop safe and effective treatments for SMA and get them approved as quickly as possible. 

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