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Qualitative analysis of patient interviews on the burden of neuronopathic Gaucher disease in Japan
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 types 2 a...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9295096/ https://www.ncbi.nlm.nih.gov/pubmed/35854314 http://dx.doi.org/10.1186/s13023-022-02429-z |