This poster was presented at the WORLDSymposium 2026 audience. Click on the poster to download a PDF of the poster.
Thought-Leadership Projects
“FDA’s inaction blocks progress for a cure” says Foundation’s O’Neill in USA Today op-ed
"Right now, 30 million Americans – half of them children – have rare diseases, 95% of which have no approved treatments. That’s why it’s so concerning that progress has been blocked by the recent rash of FDA denials of proposed treatments from drugmakers, despite the...
Foundation’s Dr. O’Neill publishes op-ed in The Hill, urging FDA to act
“The FDA must now ensure that its commitment to safety and efficacy is balanced with the urgency of patient need and the relentless progression of serious rare diseases. Action is needed now — before more life-saving therapies, and the patients who need them, fall...
Foundation launches family support webinar series, “Sanfilippo Speak”
Life with Sanfilippo Syndrome can be extremely isolating for parents and siblings. Few understand the day-to-day challenges of caring for a person with Sanfilippo Syndrome, fighting for their educational needs, and providing a safe and supportive environment. The most...
A Doctor And Mother’s Plea To FDA To Help Save Children With Rare Disease
The following opionion editorial by Dr. Cara O'Neill was published in International Business Times on Jan. 5, 2024.I'm confident that beneficial treatments for Sanfilippo exist today. We just need the FDA to break down the barrier to the accelerated approval pathway...
Sanfilippo researchers, leaders discuss data sharing to advance Sanfilippo Syndrome therapeutic development
Meeting summary now available to download Sanfilippo syndrome is an ultra-rare, neurometabolic disease which leads to severe neurodegeneration and multisystemic impacts for those affected. Individual heterogeneity, the protracted timespan of disease evolution across...
Replay of ADVANCE 2022, Sanfilippo conference, available on-demand
The virtual ADVANCE 2022, Sanfilippo Community Conference, July 7-8, 2022, brought together families and caregivers, scientists and researchers, clinicians and therapists, advocates, biotechs, and supporters. All to engage and advance the work to help children...
Registration is open for ADVANCE 2022: Sanfilippo Community Conference
Registration is open for ADVANCE 2022: Sanfilippo Community Conference! A virtual conference on July 7-8, bringing together families and caregivers, scientists and researchers, clinicians and therapists, advocates, biotechs, and supporters. All to engage and advance...
Community’s vision for Rare Disease Center for Excellence at FDA
The Speeding Therapy Access Today (STAT ACT), H.R. 1730/S. 670, includes creation of a Rare Disease Center for Excellence within the U.S. Food and Drug Administration (FDA). The legislation was introduced March 2021 and is currently assigned to the House Committee on...