Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial
# Background Pompe disease is a rare lysosomal storage disorder, leading to accumulation of glycogen characterized by muscle weakness, fatigue, pain, and, in the longer term, a requirement for ventilatory and ambulatory support, and early mortality if untreated. Clinical evidence suggests that enzym...
Saved in:
| Main Authors: | Alison Griffiths, Simon Shohet, Neil Johnson, Alasdair MacCulloch |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Columbia Data Analytics, LLC
2024-09-01
|
| Series: | Journal of Health Economics and Outcomes Research |
| Online Access: | https://doi.org/10.36469/001c.121928 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Similar Items
-
Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom
by: Lena Hubig, et al.
Published: (2023-03-01) -
Exploring Quality of Life in Adults Living With Late-onset Pompe Disease: A Combined Quantitative and Qualitative Analysis of Patient Perceptions from Australia, France, Italy, and the Netherlands
by: Holly Lumgair, et al.
Published: (2025-01-01) -
Late-Onset Pompe Disease with Nemaline Bodies
by: E. Frezza, et al.
Published: (2018-01-01) -
Late-onset Pompe disease: first clinical description in Russia
by: S. S. Nikitin, et al.
Published: (2015-02-01) -
Late-onset Pompe disease in a patient with cerebellar hemorrhage
by: Vitalii V. Goldobin, et al.
Published: (2025-04-01)