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Enzyme replacement therapy for children with acid sphingomyelinase deficiency in the real world: A single center experience in Taiwan

BACKGROUND: Acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disease with multi-systemic involvement, with no disease-modifying treatment available. Olipudase alfa is an investigational enzyme product developed to replace the deficient acid sphingomyelinase in ASMD patients. Several cl...

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Detalles Bibliográficos
Autores principales:	Pan, Yu-Wen, Tsai, Meng-Che, Yang, Chiao-Yu, Yu, Wen-Hao, Wang, Bow, Yang, Yao-Jong, Chou, Yen-Yin
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2023
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9979262/ https://www.ncbi.nlm.nih.gov/pubmed/36873248 http://dx.doi.org/10.1016/j.ymgmr.2023.100957

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