Cure Sanfilippo Foundation has published the first-ever Caregiver Preference Study for Sanfilippo Syndrome. It includes what parents consider “meaningful benefit,” as well as serves as an exploratory staging tool.
The aim of the Caregiver Preference Study is to help inform the selection and development of clinical trial endpoints to reflect desired treatment benefits across the lifespan of children with Sanfilippo Syndrome.
Publication of these findings will further inform key stakeholders, allowing the incorporation of patient voice into the decision making regarding the drug approval process and access.
The study’s project design incorporated various stakeholders, including industry partners, regulatory, and parents of children with Sanfilippo Syndrome.
More than 150 caregivers completed the quantitative survey and their response analyzed for the study.
Study results are shared via poster presentations, manuscripts, and peer-reviewed publications.
This important project has received grant support from BioMarin Pharmaceutical, Lysogene, Sobi, and Orchard Therapeutics.
Thank you to all of the authors, contributors, and parent participants who made this study possible.
Publication of Caregiver Preference & Priorities
April 2022
In April 2022, the second peer-reviewed publication from the Foundation-led Sanfilippo Caregiver Preference Study was released in the Journal of Patient Reported Outcomes.
Read the publication, “Caregivers’ assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome“.
December 2020
The first peer-reviewed publication from the Foundation-led Sanfilippo Caregiver Preference Study was released December 2020 in Neurology and Therapy.
Read the publication, “Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment“.
More about the Caregiver Preference Study
Learn more about the Caregiver Preference Study, its publication, and its presentation to the FDA.