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....
Press Release
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...
Sanfilippo parents unite on TikTok to raise $1M in 1 month for research for the deadly childhood disease
TikTok community donates more than $20,000 in 24 hours to support Sanfilippo research Several families of children with Sanfilippo Syndrome have been spreading awareness by sharing their journey with the disease and have amassed significant followings on TikTok over...
Natural History of Biomarkers & Clinical Outcomes | Type IIIA | Observational Study | Denali
Trial Information Denali Therapeutics is conducting a observational research study, which can also be called a natural history study, into Sanfilippo syndrome Type A (MPS IIIA). The purpose of the research study is to collect blood and behavioral information from...
Denali sponsoring observational research study (natural history) for Sanfilippo Type A
Denali Therapeutics has provided information about their observational research study natural history) for children with Sanfilippo Syndrome Type A. Below is a flyer provided by Denali. More information and sign-up is available here.From the study website, the purpose...
Stories that give us hope: Ryan Dant and MPS I
The above story appeared earlier this week on The Today Show, which has helped raise awareness of many families battling rare diseases including Sanfilippo. Foundation President Glenn O'Neill shared the clip on Facebook with the following message:...
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...
Help Simon update: New video sharing two miracles; one more needed
When Alina and Jeremy started sharing Simon’s story and his fight with Sanfilippo Syndrome, they hoped people would fall in love with Simon and want to help him have a chance at life. They never dreamed people would open their hearts to their entire family. When their...
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...