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

Foundation Update | New Project & Progress – May 2020

This spring hasn’t been easy for anyone. But many generous people have made it a little less stressful for families of children with Sanfilippo Syndrome (MPS III). People's support of and generosity to Cure Sanfilippo Foundation have been the rainbow in a storm of...

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Cure Sanfilippo funds $380,000 for first MPS IIIC gene therapy

Cure Sanfilippo Foundation funds $380,000 to Phoenix Nest Inc. to support path to first-ever MPS IIIC Gene Therapy COLUMBIA, SC (May 15, 2020) — Cure Sanfilippo Foundation is thrilled to announce funding of $380,000 to the New York-based biotechnology company Phoenix...

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Combined response to FDA draft guidance on drug development

On May 5, Cure Sanfilippo Foundation submitted the combined Sanfilippo Patient Advocacy Group Response to the U.S. Food And Drug Administration's (FDA) February draft guidance regarding the development of drugs to treat MPS III (Sanfilippo Syndrome). The 39-page...

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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.