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
Recent scientific articles relevant to Sanfilippo Syndrome: September 2022, digest
Every week thousands of scientific articles on various topics are published. Here are some recent articles and abstracts that are relevant to understanding, managing, and/or treating Sanfilippo Syndrome (mucopolysaccharidosis III or MPS III) as of September 2022....
Recent scientific articles relevant to Sanfilippo Syndrome: August 2022, digest
Every week thousands of scientific articles on various topics are published. Here are some recent articles and abstracts that are relevant to understanding, managing, and/or treating Sanfilippo Syndrome (mucopolysaccharidosis III or MPS III) as of August 2022....
Testing of optimized gene therapy vector shows positive results in Sanfilippo type B mice
Newly-published research, led by Professor Coy Heldermon at the University of Florida, has described the use of an optimized gene therapy vector to treat a mouse model of Sanfilippo Syndrome Type B. Researchers used an AAV8 viral delivery vector that they had...
Recent scientific articles relevant to Sanfilippo Syndrome: July 2022, digest
Every week thousands of scientific articles on various topics are published. Here are some recent articles and abstracts that are relevant to understanding, managing, and/or treating Sanfilippo Syndrome (mucopolysaccharidosis III or MPS III) as of July 2022. "Impaired...
Researchers publish results of previously-terminated Sanfilippo Type A enzyme replacement study
Study results from Sobi's previously-terminated Sanfilippo Type A enzyme replacement study have been released. The study results were published in the August 2022 edition of Molecular Genetics and Metabolism and provide some important insights. For context, this...
Sanfilippo research funding available from Foundation
Cure Sanfilippo Foundation now have three funding opportunities available for innovative research into Sanfilippo syndrome (MPS III). Cure Sanfilippo Foundation seeks to support research that fills critical gaps in current knowledge across basic science, clinical...
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.