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A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease

Background: A high prevalence of infantile-onset Pompe disease (IOPD) in the Chinese population has been noted, but there are currently no reported clinical trials of enzyme replacement therapy (ERT) for IOPD in this population. The purpose of this study was to evaluate the efficacy and safety of al...

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Detalles Bibliográficos
Autores principales:	Zhu, Diqi, Zhu, Jiacong, Qiu, Wenjuan, Wang, Benzhen, Liu, Lin, Yu, Xiaodan, Ou, Zhenheng, Shan, Guangsong, Wang, Jian, Li, Bin, Chen, Xiaokang, Liu, Cong, Li, Zipu, Fu, Lijun
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2022
Materias:	Pharmacology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9271607/ https://www.ncbi.nlm.nih.gov/pubmed/35833019 http://dx.doi.org/10.3389/fphar.2022.903488

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