We are proud to highlight the National Academies of Sciences, Engineering, and Medicine (NASEM) Consensus Study Report: Regulatory Processes for Rare Disease Drugs in the United States and European Union – Flexibilities and Collaborative Opportunities, with PPMD’s President and CEO Pat Furlong serving on the committee that helped shape its findings. This report outlines crucial steps to accelerate the development of treatments for the over 7,000 rare diseases impacting 300 million people globally—including those living with Duchenne and Becker.

The report offers key recommendations to improve regulatory processes, including:

•   Enhancing regulatory flexibility
•   Boosting clinical trial support
•   Strengthening patient engagement
•   Fostering collaboration between FDA and EMA

These recommendations reflect PPMD’s unwavering commitment to advancing policies that enhance the lives of individuals with Duchenne. By amplifying patient voices, breaking down regulatory barriers, and ensuring treatment accessibility, these efforts strive to accelerate the approval process for more patient-focused therapies. Ultimately, this means delivering effective treatments to the Duchenne and rare disease communities faster. Through the removal of obstacles and the promotion of international collaboration, we can ensure that scientific advancements become tangible hope for families confronting rare diseases.

Together, we are fighting for every future.