Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy

20260 citationsJournal Articlegold Open Access

Authors

Turner-Bowker DM · Ultragenyx Pharmaceutical (United States)

J Butler · Ultragenyx Pharmaceutical (United States)

Shayna Egan · Lumos Labs (United States)

David A. Weinstein · University of Connecticut

David Rodriguez‐Buritica · The University of Texas Health Science Center at Houston

Ayesha Ahmad · University of Michigan

María-Luz Couce

Abstract

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.

Topics & Keywords

Glycogen Storage Diseases and Myoclonus Lysosomal Storage Disorders Research Porphyrin Metabolism and Disorders

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Journal of health economics and outcomes research

Volume 13, Issue 1, pp. 39-47

DOI: 10.36469/001c.155666

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