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Read our 2025 Impact Report This Q&A is based on the December 9 webinar with Rabi Tawil, MD, and Ghinwa Dumyati, MD, from the University of Rochester Medical Center, and the January 15 webinar hosted by the National Organization of Rare Diseases with Stephen Hahn, MD, FDA commissioner

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"The 503A pathway is the first step of accountability, and I think that's really ultimately what we're looking for