Thought-Leadership Projects

Combined response to FDA draft guidance on drug development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the combined Sanfilippo Patient Advocacy Group Response to the U.S. Food And Drug Administration's (FDA) February draft guidance regarding the development of drugs to treat MPS III (Sanfilippo Syndrome).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...

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...