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Thought-Leadership Projects

Influencing the Community Working on a Cure

Curing Sanfilippo is our ultimate goal, but your support also allows us to play a critical roll in influencing the road that leads there.

The path from identifying symptoms to diagnosis to evaluating treatments needs to be as direct as possible and filled with accurate and patient-centric information for families, scientists, physicians, and regulators.

The following are projects led or supported by the Foundation that promote valuable, disease-specific and patient-preference information about Sanfilippo Syndrome, benefiting the scientific and medical communities as well as caregivers.

Projects To Influence Thinking

“Doing nothing is the biggest risk in Sanfilippo”

Sanfilippo Syndrome is ideal for the FDA's Accelerated Approval Pathway. Families are willing to accept modest benefit regarding a treatment, even if it contains risk, documents study. Glenn O’Neill closed his presentation at the 2021 Rare Disease Scientific Workshop...

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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...

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Global Genes’ RARE Cast podcast interviews Cara O’Neill

Dr. Cara O'Neill, Chief Science Officer for Cure Sanfilippo Foundation, discussed her own journey with Sanfilippo Syndrome, issues causing diagnostic delay, and how the Foundation is helping advance science for a cure in an interview as the Aug. 28 guest on Global...

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