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Long-term safety and effectiveness of velaglucerase alfa in Gaucher disease: 6-year interim analysis of a post-marketing surveillance in Japan

BACKGROUND: Gaucher disease (GD) is caused by reduced lysosomal enzyme β-glucocerebrosidase activity. Heterogeneous genotypes and phenotypes have been observed within GD types and across ethnicities. Enzyme replacement therapy is generally recommended for patients with type 1 GD, the least severe fo...

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Detalles Bibliográficos
Autores principales:	Sagara, Rieko, Ishigaki, Masahide, Otsuka, Manami, Murayama, Kei, Ida, Hiroyuki, Fernandez, Jovelle
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2021
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8642863/ https://www.ncbi.nlm.nih.gov/pubmed/34863216 http://dx.doi.org/10.1186/s13023-021-02119-2

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