Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy
Most interviewees in this open-label trial of investigational DTX401 described positive experiences, including substantial reduction in burden and improved health-related quality of life following treatment throughout the trial. To optimize patient outcomes and experience with gene therapy, guidance on and close monitoring of dietary changes during implementation should be provided.
Authors
- David Rodriguez‐Buritica (ORCID: https://orcid.org/0000-0003-1836-9853)
- David A. Weinstein (ORCID: https://orcid.org/0000-0002-1341-9367)
- Ayesha Ahmad (ORCID: https://orcid.org/0000-0003-2324-158X)
- J Butler
- Rebecca Riba‐Wolman
- Turner-Bowker DM
- Shayna Egan
- María-Luz Couce
- John J. Mitchell
Institutions
- University of Connecticut (US)
- University of Michigan (US)
- Montreal Children's Hospital (CA)
- Ultragenyx Pharmaceutical (United States) (US)
- Lumos Labs (United States) (US)
- Centre for Biomedical Network Research on Rare Diseases (ES)
- Instituto de Investigación Sanitaria de Santiago (ES)
- The University of Texas Health Science Center at Houston (US)
Publication Details
- Journal
- Journal of health economics and outcomes research
- Published
- 2026-02-12
- DOI
- https://doi.org/10.36469/001c.155666
- Primary Topic
- Glycogen Storage Diseases and Myoclonus
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Ultragenyx Pharmaceutical