Caregiver Preference Study


Dear Caregiver,

We are pleased to share with you and families who are currently living with a loved one with Sanfilippo syndrome (also known as Mucopolysaccharidosis Type III or MPS III) an opportunity to participate in an online research study.  The goal of this study is to help define what parents/caregivers feel are the most meaningful symptoms to address when developing therapies for children with Sanfilippo syndrome.  At the end of the study, collective results (not individual information) will be shared with You, the FDA, drug companies, and doctors studying Sanfilippo syndrome so that they can better incorporate the patient voice into research and drug development. We want to understand where your child is in the disease process and what is most important to YOU right now.

This is an IRB (institutional review board-ethics) approved study led by Cure Sanfilippo Foundation. De-identified survey results will be analyzed and presented to the those involved in developing treatments for Sanfilippo syndrome. No personally identifying information will be shared.

Participation in this online research survey does not prevent you from participating in any other study or research program.  This is entirely voluntary.

If you are a parent of a child who is currently living with Sanfilippo syndrome types A, B, C or D and can complete the survey in English, we hope you will click the link today to share your experience with Sanfilippo syndrome.  We would really like to hear from everyone!

Thank you for considering this important opportunity to be a voice for your children with Sanfilippo syndrome!  


Cara O’Neill, MD FAAP
Chief Science Officer, Cure Sanfilippo Foundation

For questions please contact:
Dr. Cara O’Neill at