Thought-Leadership Projects

Sanfilippo caregivers invited to take RDCRN coronavirus survey

The novel coronavirus pandemic can have a serious impact on people with rare diseases and their families. Yet, not much is known about it. The Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH), is conducting a...

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...

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...

Speeding Diagnosis of Rare Diseases By Empowering Pediatricians

First-Ever Collaboration: Pilot Program Giving Pediatricians Direct Access to Geneticists so Rare Diseases Are Diagnosed Accurately and Early Families need faster, accurate diagnosis so they can access clinical trial opportunities as soon as possible. A key to this...

Creation of Global Clinical Guidelines for Sanfilippo Syndrome

Cure Sanfilippo Foundation is leading the development the first-ever Global Clinical Guidelines for the management of Sanfilippo Syndrome in partnership with the Sanfilippo Children’s Foundation (Australia). Best-practice guidelines for clinical care are critical for...

Caregiver Preference Study for Sanfilippo Syndrome

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