Cure Sanfilippo Foundation

Come grocery shopping with the Doucette family of Saskatchewan, Canada! Find out how they navigate grocery shopping so that 5-year-old Emmett, who has Sanfilippo Syndrome Type A, can be a part of this weekly chore. Hear why it’s important that they be together as a family, what strategies Steph and Andre use to keep Emmett safe and included, and how Emmett likes to make these trips a bit exciting. Life with Sanfilippo Syndrome presents unique challenges to every aspect of life. Families of children with Sanfilippo Syndrome are generously sharing how they tackle an aspect of their life to ensure their child has the best care and quality of life. #lifewithsanfilippo #curesanfilippo

Reminder: Cure Sanfilippo Foundation is accepting Letters of Interest (LOIs) through March 28 for its ADVANCE Sanfilippo Scientific & Clinical Research Program. Cure Sanfilippo Foundation supports research across basic and translational science, clinical care, and therapeutic interventions, including clinical trials. Our overarching goal is to advance a pipeline of scientific discovery which will improve the lives of individuals with all forms of Sanfilippo syndrome. Deadline to Apply: Submit your LOI using the ProposalCentral platform by 11:00 p.m. ET on March 28, 2025. After review, selected LOIs will be invited to submit a full proposal. For more information on this current funding opportunity and priority research areas, link to submit an LOI, and to view sample forms, visit https://lnkd.in/dzXJjaH. For additional questions or to discuss your research idea with our CSO, Dr. Cara O'Neill, please contact us at [email protected].

Cure Sanfilippo Foundation Chief Science Officer and Co-Founder?Cara O'Neill O’Neill, MD, FAAP, will be a speaker at American Society of Gene & Cell Therapy’s (ASGCT) Empowering Patients 2025: A Cell and Gene Therapies Summit in March. O’Neill will join Terry Flotte, MD, University of Massachusetts for the “When Innovation Stumbles and Pathways Forward” session on March 12. The session will explore the state of commercially pre-viable therapies (sometimes referred to as “dropped” or “discontinued” trials). O’Neill will offer the perspective of a patient advocate on why this happens and potential solutions. The Empowering Patients 2025 summit is an inaugural digital event, March 12 & 13, 2025, to provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCT’s strategic plan, the goal is to empower advocates, patients, and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field, as well as patient advocacy group leaders. There will be opportunities to ask questions and deepen your knowledge of the community’s CGT journey. Registration for ASGCT’s Empowering Patients 2025 is free.?See the full agenda and/or register: https://lnkd.in/gNchxqcG

Another exciting step towards a first ever drug approval for children with Sanfilippo type A! On Feb 18, 2025, Ultragenyx announced the FDA has accepted its application seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for Sanfilippo syndrome type A (MPS IIIA). The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of August 18, 2025. No advisory committee meeting is anticipated by the FDA at this time. Read the press release from Ultragenyx: https://lnkd.in/gqngntK3

Cure Sanfilippo Foundation is now accepting Letters of Interest (LOIs) for its ADVANCE Sanfilippo Scientific & Clinical Research Program Cure Sanfilippo Foundation supports research across basic and translational science, clinical care, and therapeutic interventions, including clinical trials. Our overarching goal is to advance a pipeline of scientific discovery which will improve the lives of individuals with all forms of Sanfilippo syndrome. Deadline to Apply: Submit your LOI using the ProposalCentral platform by 11:00 p.m. ET on March 28, 2025. After review, selected LOIs will be invited to submit a full proposal. For more information on this current funding opportunity and priority research areas, link to submit an LOI, and to view sample forms, visit https://lnkd.in/dzXJjaH. For additional questions or to discuss your research idea with our CSO, Dr. Cara O'Neill, please contact us at [email protected].

Cure Sanfilippo Foundation Chief Science Officer and Co-Founder Cara O'Neill, MD, FAAP, will be a speaker at American Society of Gene & Cell Therapy’s (ASGCT) Empowering Patients 2025: A Cell and Gene Therapies Summit in March. O’Neill will join Terry Flotte, MD, University of Massachusetts for the “When Innovation Stumbles and Pathways Forward” session on March 12. The session will explore the state of commercially pre-viable therapies (sometimes referred to as “dropped” or “discontinued” trials). O’Neill will offer the perspective of a patient advocate on why this happens and potential solutions. The Empowering Patients 2025 summit is an inaugural digital event, March 12 & 13, 2025, to provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCT’s strategic plan, the goal is to empower advocates, patients, and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field, as well as patient advocacy group leaders. There will be opportunities to ask questions and deepen your knowledge of the community’s CGT journey. Registration for ASGCT’s Empowering Patients 2025 is free. See the full agenda and/or register: https://lnkd.in/gNchxqcG

How can ABA therapy be used when a patient has a degenerative disease like Sanfilippo Syndrome? Here's what licensed ABA therapist Caroline Roesel, M.Ed., BCBA, LBA, had to say during last week’s Sanfilippo Speak. Watch the complete episode on tips & tricks for insurance & ABA therapy for patients with Sanfilippo Syndrome at https://lnkd.in/g_RdVNcS.

We are headed to WORLDSymposium 2025 next week to spend valuable time at the largest annual gathering of lysosomal disease experts from around the globe. Since Eliza's diagnosis in 2013, we have attended WORLD every year. The value of engaging with the world's leading experts has led to some fantastic collaborations and partnerships. Additionally, the opportunity to meet with other patient advocacy group leaders is absolutely invaluable. If you are at WORLD, we hope to see you! We'll be in the expo hall (booth 609), as well as at sessions and events. Glenn O'Neill Cara O'Neill

Today at 1:00 p.m. EST, explore tips and tricks for Applied Behavioral Analysis (ABA) for individuals with Sanfilippo Syndrome and navigating insurance for it. Our ABA therapist will discuss long-term planning, goal setting, and insurance challenges in ABA therapy for children with Sanfilippo Syndrome. Open to everyone. To join, simply follow the Zoom link at 1:00 p.m.: Zoom link to join the webinar: https://lnkd.in/gKyS3RSW We look forward to seeing you there! Unable to join live? A recording of the webinar will be available on the Sanfilippo Speak page of the Foundation’s website afterward for on-demand access.