Dr. Cara O'NeillChief Science Officer for Cure Sanfilippo Foundation The pandemic has Cure Sanfilippo Foundation embarking on some new advocacy work by Chief Science Officer Cara O'Neill. Many, many children with significant special needs, especially children with...
Collaboration
Cure Sanfilippo joins in calling for addition to Cures 2.0 legislation
Cure Sanfilippo Foundation is one of 46 patient advocacy organizations to sign-on to a letter asking U.S. Representatives Diana DeGette (D-Colorado) and Fred Upton (R-Michigan) to include H.R. 4144 - Ending the Diagnostic Odyssey Act in the Cures 2.0 legislation. The...
Foundation contributes to published list of early symptoms of MPS III
Cure Sanfilippo Foundation Chief Science Officer Cara O'Neill, MD, FAAP, and Board Member Valerie Byers, PhD, are among the contributors of a newly-published paper that identifies the most-important, early symptoms of Mucopolysaccharidosis III (MPS III) to assist...
Key Points of Sanfilippo Advocacy Group Response to FDA Guidance
The combined Sanfilippo Advocacy group submitted comments to the U.S. Food and Drug Administration's (FDA) on May 5, 2020, regarding its draft guidance on drug development for Sanfilippo Syndrome for review. These advocacy group is working closely with the FDA on the...
Cure Sanfilippo funds $380,000 for first MPS IIIC gene therapy
Cure Sanfilippo Foundation funds $380,000 to Phoenix Nest Inc. to support path to first-ever MPS IIIC Gene Therapy COLUMBIA, SC (May 15, 2020) — Cure Sanfilippo Foundation is thrilled to announce funding of $380,000 to the New York-based biotechnology company Phoenix...
Combined response to FDA draft guidance on drug development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the combined Sanfilippo Patient Advocacy Group Response to the U.S. Food And Drug Administration's (FDA) February draft guidance regarding the development of drugs to treat MPS III (Sanfilippo Syndrome).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...
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...
Parent Prioritization of Meaningful Treatment Targets for Sanfilippo Syndrome
At the WORLD Symposium 2020, Cure Sanfilippo Foundation Chief Science Officer Cara O'Neill, MD, FAAP, and Research Projects Coordinator Elise Drake, PhD, gave a poster presentation entitled "Parent Prioritization of Meaningful Treatment Targets for Sanfilippo...