Thought-Leadership Projects

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...

“What is a life worth?” is an unconscious bias among clinicians

Dr. Cara O'Neill is Chief Science Officer for Cure Sanfilippo Foundation and mother to a 10-year-old daughter with Sanfilippo Syndrome. During a wide-ranging interview with Sonia Adnan of Instagram's autismlifegram.md, they explored the mentality toward treating...

Combined response to FDA draft guidance on drug development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the combined Sanfilippo Patient Advocacy Group Response to the U.S. Food And Drug Administration's (FDA) February draft guidance regarding the development of drugs to treat MPS III (Sanfilippo Syndrome).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