Association between under-dose of enzyme replacement therapy and quality of life in adults with late-onset Pompe disease in China: A retrospective matched cohort study
A Pompe-disease specific instrument based on patient experiences is warranted to closely monitor changes in QoL on a routine basis. It is desirable for patients with severe under-dose ERT to discuss with physicians whether to adjust treatment strategies.
Authors
- Richard Huan Xu (ORCID: https://orcid.org/0000-0002-4720-5172)
- Jiazhou Yu (ORCID: https://orcid.org/0000-0002-9249-2721)
- Shanquan Chen (ORCID: https://orcid.org/0000-0002-4724-4892)
- Dong Dong (ORCID: https://orcid.org/0000-0001-9784-6472)
- Huanyu Zhang (ORCID: https://orcid.org/0000-0001-6480-2510)
- Siyue Yu (ORCID: https://orcid.org/0000-0001-7360-2406)
Institutions
- Hong Kong Polytechnic University (HK)
- Sun Yat-sen University (CN)
- Chinese University of Hong Kong (HK)
- London School of Hygiene & Tropical Medicine (GB)
- The Seventh Affiliated Hospital of Sun Yat-sen University (CN)
- Hong Kong Jockey Club (HK)
- Chinese University of Hong Kong, Shenzhen (CN)
Publication Details
- Journal
- PLoS ONE
- Published
- 2024-09-17
- DOI
- https://doi.org/10.1371/journal.pone.0310534
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.33