The 21st Century Cures 2.0 Act legislation from U.S. Representatives Diana DeGette (D-Colorado) and Fred Upton (R-Michigan) could have a profound impact on the speed of therapy development and diagnostic access for patients with rare diseases. "Many organizations have...
Collaboration
Cure Sanfilippo agrees: The need to use surrogate biomarkers in an accelerated drug approval pathway for diseases like Sanfilippo
A powerful article from Dr. Emil D. Kakkis outlines the critical need for use of surrogate biomarkers in an accelerated drug approval pathway for diseases like Sanfilippo syndrome. In the article, "Aduhelm’s accelerated approval offers a promising roadmap for rare...
Research: Lysosomal storage disorders with altered heparan sulfate metabolism lead to dopamine-dependent autistic behaviors in Sanfilippo mouse models
A research project co-funded by Cure Sanfilippo Foundation and Sanfilippo Children's Foundation (Australia) recently published its findings, "Altered heparan sulfate metabolism during development triggers dopamine-dependent autistic-behaviours in models of lysosomal...
History being made: ScreenPlus, the largest U.S. newborn screening study, includes Sanfilippo
History is being made! Starting this week, newborns in the largest ever U.S. newborn screening study, ScreenPlus, are being screened for Sanfilippo syndrome Type B! Addition of Sanfilippo Type A will follow in the coming months! Why invest in Newborn Screening...
Cure Sanfilippo supports North Carolina legislation HB 736 to expand state newborn screening
Cure Sanfilippo Foundation has joined other rare disease patient organizations in thanking the North Carolina House of Representatives for its leadership in passing HB 736, Timely Updates to Newborn Screening Program, and calling for the Senate to also pass the...
Cure Sanfilippo joins in calling Congress to support The BENEFIT Act
Cure Sanfilippo Foundation has signed-on to a stakeholder letter that calls for the U.S. Congress to support the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act (S. 373), sponsored by U.S. Senators Roger Wicker (R-MS) and Amy Klobuchar...
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...
Foundation now accepting LOIs for Sanfilippo research funding
Cure Sanfilippo Foundation is now accepting Letters of Intent (LOI) 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 care, and...
2nd annual World Sanfilippo Awareness Day to be celebrated Nov. 16, 2020
People around the world are making a significant difference for children with Sanfilippo Syndrome. Each day, an effective, approved, and available treatment for Sanfilippo Syndrome is closer. Each year, World Sanfilippo Awareness Day is celebrated by this community...