Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease.

Laura E Case,Carl Bjartmar,Claire Morgan,Robin Casey,Joel Charrow,John P Clancy,Majed Dasouki,Stephanie DeArmey,Khan Nedd,Mary Nevins,Heidi Peters,Dawn Phillips,Zachary Spigelman,Cynthia Tifft,Priya S Kishnani

Neuromuscular Disorders（2015）

引用 45|浏览9

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摘要

•Increased ERT dose may be beneficial in Pompe patients with plateau/decline in motor function.•Improvements in functional mobility were observed.•Improvements in fine motor skills are seen in these patients.•Dose adjustments during plateau/decline are important because disease progression can occur.

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关键词

Alglucosidase alfa,Enzyme replacement therapy,Infantile onset,Late onset,Pompe disease,Clinical decline,Dose

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