Science & Research News

What is Happening In Sanfilippo Research?

Stay up to date on the latest information regarding scientific aspects of Sanfilippo Syndrome and progress to find a treatment or cure.

Recent News

JR-441 Enzyme Replacement | Type IIIA | Phase I-II | JCR

Page last updated: October 23, 2023 For web accessibility options: Click/tap the floating blue icon on the right. Trial Information Phase I/​II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA A Phase I/ II, open-label, randomized, 2-arm study,...

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Jessica Haywood joins Cure Sanfilippo Foundation board

Jessica Haywood has joined the board for Cure Sanfilippo Foundation, bringing additional data analytics and social media expertise, strategic thinking, and the patient voice and lived experience of life with Sanfilippo Syndrome from a close relative’s perspective. “We...

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Other Sources of News

ClinicalTrials.gov

Immediately see when clinical trials for Sanfilippo Syndrome are listed and detailed information.

Sanfilippo News

A site that aggregates news about Sanfilippo Syndrome into a single location.

RARE Cast – Global Genes’ Podcast

Hosted by award-winning journalist Daniel Levine, RARE Cast focuses on the intersection of rare disease with business, science, and policy in a weekly podcast.