YOUR donations social shares, and outreach to congressional representatives helped get the first ever treatment for Sanfilippo syndrome approved!
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Welcome to Hope for Merrick!
This page is dedicated to raising awareness and funds to support research for a cure for Sanfilippo Syndrome, a rare and devastating neurodegenerative disease affecting our beloved son, Merrick, and many other children around the world.
Merrick is a joyful child who loves puzzles, monster trucks, and Transformers. Despite his abundant energy and love for life, Merrick faces immense challenges due to Sanfilippo Syndrome, often likened to Alzheimer's in children. This heartbreaking condition leads to progressive mental and physical decline.
Our mission is to support the Cure Sanfilippo Foundation's efforts in funding critical research and clinical trials aimed at finding cures for every type of this disease. Through our fundraising initiatives, especially our "Cure Merrick" campaigns, community engagement, and spreading awareness, we aim to bring hope and action together to make a difference in Merrick's life and the lives of countless other children battling this disease.
Join us on this journey of hope and support. Together, we can make progress towards a cure for all children affected by Sanfilippo Syndrome.
Thank you for your love, support, and generous contributions.
With gratitude, Nancy, Matt and Merrick
Ways to support Merrick
Ways to support Merrick
Updates
Updates
Update as of September 17, 2026
Update as of September 17, 2026
FDA approves first-ever treatment for Sanfilippo Syndrome Type A, FAYUVI™
On September 17, 2026, the U.S. Food and Drug Administration (FDA) granted standard full approval of FAYUVI™ (rebisufligene etisparvovec-hopf), also known as UX111, for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A).
FAYUVI is the first-ever FDA-approved treatment for Sanfilippo syndrome Type A, a progressive and fatal neurodegenerative disease.
“For families living with Sanfilippo syndrome type A, the trajectory of this disease is heartbreaking — children who develop normally in their earliest years facing a relentless regression with no approved treatment to slow it. Parents and clinicians have been waiting far too long for an option,” said Karim Mikhail, B. Pharm., M.S., Director of the Center for Biologics Evaluation and Research in FDA’s announcement. “Today’s approval of Fayuvi is a meaningful step forward — not only for these children and their families, but for the promise of gene therapy to address rare and devastating diseases where the need for safe and effective treatment is the most urgent.”
Update as of February 26, 2026
Update as of February 26, 2026
Cure Sanfilippo Foundation Chief Science Officer Cara O’Neill, MD, was among four expert witnesses testifying before the U.S. Senate Special Committee on Aging to explore how U.S. Food and Drug Administration processes and evolving standards unintentionally delay patient access to safe and effective therapies, particularly for individuals living with rare diseases.
“Congress has given FDA the tools of flexibility it needs to accelerate approvals for these devastating diseases. But sadly, flexibility and speed are not actually what most rare disease patients are witnessing. Transformative therapies are at FDA's doorstep. And so with great respect, families are pleading for FDA to unlock the door and move with urgency so that our children can have a chance at the life they deserve.”
- Dr. Cara O’Neill, Chief Science Officer and Co-Founder, Cure Sanfilippo Foundation, and mother of Eliza (age 16)
Other ways to support
Other ways to support
Make purchases through our Cure Sanfilippo store, Amazon page, or TikTok showcase
All commission earned will be donated to the Cure Sanfilippo Foundation.
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