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Thought-Leadership Projects

Community’s vision for Rare Disease Center for Excellence at FDA

The Speeding Therapy Access Today (STAT ACT), H.R. 1730/S. 670, includes creation of a Rare Disease Center for Excellence within the U.S. Food and Drug Administration (FDA). The legislation was introduced March 2021 and is currently assigned to the House Committee on...

“Doing nothing is the biggest risk in Sanfilippo”

Sanfilippo Syndrome is ideal for the FDA's Accelerated Approval Pathway. Families are willing to accept modest benefit regarding a treatment, even if it contains risk, documents study. Glenn O’Neill closed his presentation at the 2021 Rare Disease Scientific Workshop...