As you know, ensuring appropriate federal funding for Duchenne research has been a cornerstone of PPMD’s advocacy agenda over the past decade. Our persistence – your persistent and collective voice - has paid off with over $250 million directed to Duchenne research.
Today we now find ourselves at a pivotal moment with multiple potential therapies for Duchenne in various stages of the development process. We must now, once again, look forward to what is next for Duchenne.
While we will remain unwavering in our requests for increased Duchenne research funding, our focus must now have an increased attention on the Food and Drug Administration (FDA). We must make sure this agency has the expertise and tools necessary to accurately review and evaluate Duchenne candidate therapies in the quickest amount of time possible to ultimately deliver safe and effective therapies to our children as quickly as possible.
At this time, Congress is working on writing a critical FDA related bill called the Prescription Drug User Fee Act (PDUFA), and PPMD is working with other rare disease advocates on provisions to benefit our sons. Earlier this year, the PPMD Board of Directors adopted a policy position that, among other things, seeks to ensure the potential benefits of a therapy are weighted appropriately against possible risks.
PPMD is working now to develop a specific legislative provision or provisions that would advance these core principles, and will be committed to working this issue as Congress writes its FDA bill over the coming months.
Please take a moment to read this policy. We cannot stress enough how important it is for your family, for every Duchenne family and organization to understand this topic, as it will most definitely impact how quickly treatments are available to our sons.
Knowledge is Power
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