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...
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...
Combined response to FDA draft guidance on drug development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the combined Sanfilippo Patient Advocacy Group Response to the U.S. Food And Drug Administration's (FDA) February draft guidance regarding the development of drugs to treat MPS III (Sanfilippo Syndrome).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...
Sanfilippo caregivers invited to take RDCRN coronavirus survey
The novel coronavirus pandemic can have a serious impact on people with rare diseases and their families. Yet, not much is known about it. The Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH), is conducting a...
Survey: Caring For A Child With Rare Genetic Disorder During COVID-19
Families with a rare genetic disorder such as Sanfilippo Syndrome will have a different experience than others during the COVID-19 emergency. Scientists at UCLA are working directly with families to better understand specific challenges and when they occur by...
TIGEM identifies CLR01 as new potent drug candidate for MPS IIIA
Dr. Fraldi and his team at TIGEM (Telethon Institute of Genetics and Medicine) in Italy have been working to identify a new treatment strategy for neurodegenerative lysosomal storage diseases. Cure Sanfilippo Foundation has supported this study thanks to the support...
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.