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Collaboration

Sanfilippo research funding available from Foundation

Cure Sanfilippo Foundation now have three funding opportunities available for innovative research into Sanfilippo syndrome (MPS III). Cure Sanfilippo Foundation seeks to support research that fills critical gaps in current knowledge across basic science, clinical...

Replay of ADVANCE 2022, Sanfilippo conference, available on-demand

The virtual ADVANCE 2022, Sanfilippo Community Conference, July 7-8, 2022, brought together families and caregivers, scientists and researchers, clinicians and therapists, advocates, biotechs, and supporters. All to engage and advance the work to help children...

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...