Advocacy

The policies that federal and state legislatures debate and the laws they pass affect nearly all aspects of life for those affected by SMA.

Cure SMA works diligently to monitor legislative and regulatory happenings in Washinton, DC and across the country to ensure that policies and programs are responsive to and reflect the needs of individuals with SMA and their families. Our team works with public policy experts and advocates in Washington and in the state capitals to keep abreast of developments impacting you and your family. 

Some of the issues we are watching include:

  • Federal funding for vital biomedical research
  • Policies, funding, and programs related to newborn screening, healthcare coverage, and Medicaid eligibility requirements
  • Making determinations on special education funding and policies

Legislative Agenda

In addition to work on the above issues, Cure SMA is actively engaged on three primary legislative goals:

  • Working with Congress and the FDA to expedite the drug-approval process for SMA therapies and help ensure that treatments reach patients in the fastest and safest manner possible
  • Implementing a national program to screen every newborn for SMA to ensure that babies with SMA are identified at the earliest possible moment so treatment can begin and any adverse impact of the disease can be minimized
  • Increasing federal funding for SMA research to ensure we continue to make progress in identifying effective treatments and care for all types of SMA. 

Newborn Screening

In May 2017, SMA was unanimously accepted into the evidence review stage for the federal Recommended Uniform Screening Panel, with a decision expected in six to nine months. Adding SMA to the RUSP is a critical step toward our goal of having SMA added to newborn screening programs across the US.

However, the final decision on implementation of newborn screening ultimately rests with each state, and each state has its own process for implementation, meaning we will need “SMA champions” in all 50 states to help advocate on behalf of our community. While a successful RUSP application will strongly support our case for implementation, in most states we do not need to wait for a decision in order to begin the process. The resources below are provided to assist our community as we look to effectively advocate for newborn screening across the US.

State advocacy and other newborn screening activities are supported by the SMA Newborn Screening Coalition. Members of the SMA Newborn Screening Coalition include representatives from pharmaceutical companies Biogen and AveXis, and staff members from Cure SMA.

Resources

Use the following links to download these resources.

Toolkit and Handouts

State Fact Sheets

To request paper copies of any of these materials, or to request a state fact sheet not shown here, email advocacy@curesma.org.

Effective Advocacy

Direct communication with the leadership of Congress and Federal Agencies is critically important to achieving each of our goals. Cure SMA is continuously working to increase its presence within the U.S. House of Representatives and the U.S. Senate as well as the various health related agencies within the federal government, including the National Institutes of Health (NIH), the Health Resources and Services Administration (HRSA), the Centers for Disease Control and Prevention (CDC), and the Centers for Medicare and Medicaid Services (CMS).  In addition to the work we do within Washington, DC on your behalf, your support and participation are critical to the success of these efforts. 

Cure SMA also works in partnership with other patient advocacy and health professional groups to help promote the importance of our issues, leveraging resources, and voicing our concerns and making our requests of policymakers in a collective manner. Working collaboratively with others who share our goals helps us increase our effectiveness. 

Getting Involved

Our news section includes many recent legislative alerts. However, you can also reach out to your Representatives or Senators to voice your concerns on any number of topics important to the SMA community.

Our Targets 

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