Advocacy Year-in-Review 2018

For Cure SMA, 2018 has been a year of great progress. As we continue to make strides into 2019, it is important to take a moment to look back at all our successes. Thank you to all our families and advocates for your support and continued efforts to cure SMA. We wish you all a very Happy Holiday!

Highlights of 2018

SMA Added to the Recommended Uniform Screening Panel

After tremendous work from the SMA community, Health and Human Services Secretary Alex Azar approved the recommendation to add SMA to the Recommended Uniform Screening Panel (RUSP) for newborn screening in July. The RUSP is a list of 35 core conditions that experts in science, medicine, genetics and health policy agree each state should test for in its newborn screening programs. This was a significant milestone, since it is a scientific stamp of approval for screening newborns for SMA. Many states follow the RUSP for their newborn screening and others will not add a condition until it is added to the RUSP.

SMA Newborn Screening across the United States

A major focus this year has been on including SMA in states’ newborn screening panels. Although getting on the RUSP was a major victory, we now must get each state to add on the Secretary’s recommendation and start screening for SMA. We know that in order to give SMA patients the very best treatment, we need to diagnose the condition as soon as possible, which may be long before they start showing symptoms. Newborn screening is the most efficient and effective way to do this. Currently, Utah, Minnesota and New York all screen for SMA. Wisconsin, North Carolina and Massachusetts are currently running temporary SMA screening programs and we expect Georgia to begin very soon. The temporary programs help these states to understand what a large-scale state-wide screening for SMA would look like in practice. These states will use what they learn in these temporary programs to develop permanent ones. Kansas, Missouri, Illinois, Indiana, Michigan, Ohio, Pennsylvania, Maryland, Virginia and New Hampshire have adopted SMA screening but have not yet implemented it, meaning that they have decided to do this screening, but are still implementing the process. Several more states are in the process of adding SMA to their panels. We expect even more states to begin their screening in 2019.

Increase in Funding

We are excited to announce that this year, we saw an increase in federal funding for both biomedical research and newborn screening. The federal government directed an additional $5 billion to biomedical research through the National Institutes of Health, including the centers there that focus on neurological disorders and child development. An additional $2 million went to programs that help states purchase new equipment for their newborn screening labs, train staff and ensure scientific accuracy in their testing.

Gene Therapy Submitted to FDA

This December, Novartis announced that the U.S. Food and Drug Administration (FDA) accepted their Biologics License Application (BLA), a request to introduce and deliver a biologic product within the states and has been granted Priority Review by the FDA for their gene replacement therapy drug ZOLGENSMA. Priority review means, that the FDA will review the application within six months instead of ten months, expediting the process. This groundbreaking new treatment is showing incredible promise in asymptomatic patients in clinical trials.

Looking Ahead to 2019

The new year will bring even more exciting news and advancements for the Cure SMA community!

Newborn Screening Saves Lives Reauthorization Act

In January, the Newborn Screening Saves Lives Reauthorization Act will be reintroduced to the new Congress. The Newborn Screening Saves Lives Reauthorization Act will renew federal programs that support each state’s newborn screening programs. It will also continue funding so that state’s can enhance their newborn screen programs to ensure that all newborns are screened for conditions and receiving the best quality of care and follow-up. Stay tuned for how you and your family can get involved in advocating for this important legislation!

Rare Disease Day

February 28th is Rare Disease Day. Cure SMA will be participating in advocacy efforts on behalf of the SMA community and other rare conditions. There will be more information on actions you and your family can take to come in the new year! Check out and learn more about Rare Disease Day here.

Gene Therapy on the Horizon

We expect the Food and Drug Administration (FDA) to approve ZOLGENSMA, the gene replacement therapy made by Novartis and Avexis later this year. Once this happens, we’ll provide information about the drug, who may be eligible and will continue to advocate to ensure access to the best treatments.

Addition of SMA Newborn Screening in Several States

Lastly, in the new year several states, including Indiana, Missouri and Maryland, will begin screening newborns for SMA, and several other states will also move forward in the process for adding SMA to their newborn screening panels. This will bring us closer to our goal of making sure every newborn across all 50 states is screened for SMA.

Through advocacy and policy, Cure SMA continues to empower our community and give a voice to individuals and families that are faced with the daily challenges of SMA. We work with legislators, regulators and officials in Washington D.C. and in the states to advocate on behalf of our community. If you have any questions, stories, ideas or concerns that are related to advocacy and policy, please feel free to send us a message at [email protected]. Your voice and participation in advocacy are critical to our success.

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