The Sept. 7, 2021, episode of the Foundation's "A Close Look at Sanfilippo" podcast spends 10 minutes (and a little more in this episode) diving into five hot topics regarding Sanfilippo Syndrome, a rapidly degenerative disease in children currently with no...
Scientific News
Podcast Episode 3: Free educator guide; Dopamine & reversing Autism behaviors + more
The Aug. 23, 2021, episode of the Foundation's "A Close Look at Sanfilippo" podcast spends 10 minutes (and a little more in this episode) diving into five hot topics regarding Sanfilippo Syndrome, a rapidly degenerative disease in children currently with no...
Advancing the Understanding of Special Education and Therapeutic Needs of Children with Neurodegenerative Disorders
The challenges of the pandemic shined a light on the critical role of special educators and rehabilitative therapists to protect the skills of children’ with neurodegenerative disorders. This academic publication and white paper provides teachers with a guide for...
Podcast Episode 2: In Memory of Reagan; New testing created; $1M granted for 5 new projects + more
It's been just two weeks since the last episode of the Foundation's "A Close Look at Sanfilippo" podcast, and there's already so much to share! The podcast's 10 minutes dives into: The life and importance of Reagan McGee; How your support of the Foundation helped...
New enzyme assay testing method for Sanfilippo created with Foundation funding
A New and Improved Testing Option for Evaluating Potential Drugs for Sanfilippo In the fight to cure Sanfilippo syndrome, a key basic requirement is to be able to measure critical substances and to do so as accurately, easily, and inexpensively as possible. The study...
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...
Foundation raises nearly $4 million with three GoFundMe campaigns to cure Sanfilippo Syndrome
Current campaign is nearing the $1 million mark to fund a first-ever clinical trial Cure Sanfilippo Foundation is on track to raise another $1 million via a GoFundMe fundraiser for research and clinical trials to cure Sanfilippo Syndrome. This is the third...
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...