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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...

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Detalles Bibliográficos
Autores principales: Koto, Yuta, Narita, Aya, Noto, Shinichi, Ono, Midori, Hamada, Anna Lissa, Sakai, Norio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
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