Qualitative analysis of patient interviews on the burden of neuronopathic Gaucher disease in Japan
Abstract Background Gaucher disease (GD) is a rare, autosomal recessive lysosomal storage disorder that adversely affects life expectancy and health-related quality of life (HRQOL). Although HRQOL questionnaires are available for type 1 GD, they are not suitable for patients with the neuronopathic t...
Main Authors: | , , , , , |
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Format: | Article |
Language: | English |
Published: |
BMC
2022-07-01
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Series: | Orphanet Journal of Rare Diseases |
Subjects: | |
Online Access: | https://doi.org/10.1186/s13023-022-02429-z |