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

Combined response to FDA draft guidance on drug development

On May 5, Cure Sanfilippo Foundation submitted the combined Sanfilippo Patient Advocacy Group Response to the U.S. Food And Drug Administration's (FDA) February draft guidance regarding the development of drugs to treat MPS III (Sanfilippo Syndrome). The 39-page...

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What does “better” mean and who gets to decide that?

Dr. Cara O'Neill, Chief Science Officer for Cure Sanfilippo Foundation, posed this question regarding study endpoints to an audience of before the audience of research scientists, biotech leads and industry partners, physicians, U.S. Food & Drug Administration...

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Caregiver Preference Study for Sanfilippo presented to FDA

The urgent mission to save children from the fatal and rapidly-degenerative disease Sanfilippo Syndrome must continue because the disease's devastating effects never stop, no matter what else is happening in the world. With this mindset, Cure Sanfilippo Foundation and...

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Caregiver Preference & Priorities for Sanfilippo Syndrome

Cure Sanfilippo Foundation has published the first-ever Caregiver Preference Study for Sanfilippo Syndrome. It includes what parents consider “meaningful benefit,” as well as serves as an exploratory staging tool. The aim of the Caregiver Preference Study is to help...

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