Caregiver Preference Study for Sanfilippo Syndrome

March 1, 2019

Cure Sanfilippo Foundation is publishing 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 project design for the Caregiver Preference Study 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.

Publication of Caregiver Preference Study

The first peer-reviewed publication from the Foundation-led Sanfilippo Caregiver Preference Study was released December 2020 in Neurology and Therapy.

Thank you to all of the authors, contributors, and parent participants who made this study possible.

Read the publication, “Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment“.

Poster Presentations of Caregiver Preference Study

Outcomes and interim results from the study’s focus groups (the qualitative portion) were shared in a Platform and Poster Presentation at the World Symposium in February 2019, and can be found here: presentation poster from the WORLD Symposium 2019.

Cara O'Neill presenting Caregiver Preference Study results at national conference

Dr. Cara O’Neill, Chief Science Officer for Cure Sanfilippo Foundation, presenting a poster on the caregiver preference study at WORLD Symposium 2019.

Sanfilippo Syndrome caregiver preference study poster presentation

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