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Long-Term Observation of the Safety and Effectiveness of Enzyme Replacement Therapy in Japanese Patients with Pompe Disease: Results From the Post-marketing Surveillance

INTRODUCTION: Alglucosidase alfa received marketing approval for the treatment of Pompe disease in Japan in 2007. We conducted a post-marketing surveillance study to monitor the long-term safety and efficacy of alglucosidase alfa therapy among Japanese patients with Pompe disease. METHODS: The safet...

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Publicat a:Neurol Ther
Autors principals: Nagura, Hitoshi, Hokugo, Jiro, Ueda, Kazuo
Format: Artigo
Idioma:Inglês
Publicat: Springer Healthcare 2019
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6858897/
https://ncbi.nlm.nih.gov/pubmed/31559584
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40120-019-00157-4
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