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
VIDEO: What Craig & Sara want to say to you
https://youtu.be/dOxewsJad4w This is Craig and Sara. Their beautiful son Carson is participating in a new clinical trial that your generosity made happen. This is their message to you. Your support enabled Cure Sanfilippo Foundation to build this clinical trial...
Cure Sanfilippo joins in calling for addition to Cures 2.0 legislation
Cure Sanfilippo Foundation is one of 46 patient advocacy organizations to sign-on to a letter asking U.S. Representatives Diana DeGette (D-Colorado) and Fred Upton (R-Michigan) to include H.R. 4144 - Ending the Diagnostic Odyssey Act in the Cures 2.0 legislation. The...
Foundation contributes to published list of early symptoms of MPS III
Cure Sanfilippo Foundation Chief Science Officer Cara O'Neill, MD, FAAP, and Board Member Valerie Byers, PhD, are among the contributors of a newly-published paper that identifies the most-important, early symptoms of Mucopolysaccharidosis III (MPS III) to assist...
Cure Sanfilippo expands its website to provide additional information
Information, support, and resources regarding the more than 7,000 rare diseases in the world, which includes Sanfilippo Syndrome, can be scarce. Cure Sanfilippo Foundation has expanded its website to help fill the gap of information about Sanfilippo Syndrome for...
Key Points of Sanfilippo Advocacy Group Response to FDA Guidance
The combined Sanfilippo Advocacy group submitted comments to the U.S. Food and Drug Administration's (FDA) on May 5, 2020, regarding its draft guidance on drug development for Sanfilippo Syndrome for review. These advocacy group is working closely with the FDA on the...
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...
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.