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

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

Abstract 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 Th...

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Bibliographic Details
Main Authors: Hitoshi Nagura, Jiro Hokugo, Kazuo Ueda
Format: Article
Language:English
Published: Adis, Springer Healthcare 2019-09-01
Series:Neurology and Therapy
Subjects:
Alglucosidase alfa
Clinical outcome
Enzyme replacement therapy
Pompe disease
Post-marketing surveillance
Japanese cohort
Online Access:http://link.springer.com/article/10.1007/s40120-019-00157-4

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