Development and evaluation of a patient-reported outcome measure specific for Gaucher disease with or without neurological symptoms in Japan

Abstract Background Patients with Gaucher disease (GD), a rare lysosomal storage disorder, have reduced health-related quality of life (HRQOL). A patient-reported outcome measure (PROM) for HRQOL developed for type 1 GD (GD1) is not appropriate for patients with neuronopathic GD (nGD) types 2 (GD2)...

ver descrição completa

Detalhes bibliográficos

Principais autores:	Aya Narita, Yuta Koto, Shinichi Noto, Masafumi Okada, Midori Ono, Terumi Baba, Rieko Sagara, Norio Sakai
Formato:	Artigo
Idioma:	Inglês
Publicado em:	BMC 2024-01-01
Colecção:	Orphanet Journal of Rare Diseases
Assuntos:	Gaucher disease Japan Patient reported outcome measures Quality of life Burden of disease Neuronopathic Gaucher disease
Acesso em linha:	https://doi.org/10.1186/s13023-023-02996-9
Tags:	Adicionar Tag Sem tags, seja o primeiro a adicionar uma tag!