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...

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Auteurs principaux:	Yuta Koto, Aya Narita, Shinichi Noto, Midori Ono, Anna Lissa Hamada, Norio Sakai
Format:	Artigo
Langue:	Inglês
Publié:	BMC 2022-07-01
Collection:	Orphanet Journal of Rare Diseases
Sujets:	Gaucher disease Interviews as topic Patient reported outcome measures Qualitative research Burden of disease Japanese
Accès en ligne:	https://doi.org/10.1186/s13023-022-02429-z
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