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Support the Development of Treatments for Pediatric Rare Disease

For children living with a neuromuscular disease, access to safe and effective treatments is of vital importance – neuromuscular diseases can advance quickly, and without timely access to new treatments, children and their families lose precious time while waiting for these treatments to become available. 

There is a critical program, known as the Rare Pediatric Disease Priority Review Voucher Program, that helps solve this challenge. The program incentivizes drug companies to develop treatments for rare pediatric diseases by offering a priority review voucher for future drug applications that a company submits to the Food and Drug Administration (FDA). This is a valuable incentive, bringing potentially life-saving treatments to the market – and to families – faster. 

However, this program will expire this week if Congress does not reauthorize it. You can help us make sure that they do! 

Reach out to your Members of Congress to urge them to support the end-of-year Continuing Resolution that contains a reauthorization of the Rare Pediatric Disease Priority Review Voucher Program. We need your support today to ensure that the voucher program is reauthorized before it expires. You can also share your story with Congress to express how this program has impacted you or a loved one. Feel free to share this action alert with others who may share similar experiences.

Join us and support this critical reauthorization today!

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