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
Newborn screening pilot ScreenPlus that includes Sanfilippo Type A and B
When children with a rare disease are diagnosed at birth, they have the greatest opportunity to receive timely, approved treatments or participate in clinical trials that can provide them better quality of life and less pain and suffering. Early diagnosis and...
Strengthening the rationale for the use of the “molecular tweezer” CLR01 in the treatment of Sanfilippo
Cure Sanfilippo Foundation has co-funded a research grant with Sanfilippo Children's Foundation (Australia) and H.A.N.D.S consortium, an international network of Type C families and groups. This grant is to Associate Professor Alessandro Fraldi at CEINGE - Advanced...
Free online CME course available on Sanfilippo syndrome
A free, online, continuing medical education (CME) course is available for any healthcare provider interested in learning about Sanfilippo syndrome. The 30-minute course is targeted to healthcare providers who are unfamiliar with Sanfilippo, also known as...
New study: Economic burden of rare diseases estimated at $966B in 2019
In the most-comprehensive assessment of the total cost of rare diseases in the U.S. to date, the EveryLife Foundation for Rare Diseases' estimates in its "The National Economic Burden of Rare Disease Study" that the estimated economic cost of 379 rare diseases in the...
Lysogene shares report on LYS-SAF302 biomarker data
Lysogene shared the following press release regarding its LYS-SAF302 Biomarker Data. The results were first shared at WORLDSymposium 2021 in February.PRESS RELEASE Lysogene Reports LYS-SAF302 Biomarker Data Presented at the WORLDSymposium™ 2021 Evidence of positive...
Investigating NDST1 inhibitors as target for substrate reduction therapy for Sanfilippo
Identifying a novel class of substrate reduction therapy drugs for the mucopolysaccharidoses that inhibit N-deacetylase/N-sulfo-transferase (NDST) Cure Sanfilippo Foundation has co-funded a one-year research project with Sanfilippo Children's Foundation (Australia) to...
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.