Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report.

Priya S Kishnani,David Kronn,Anaïs Brassier,Alexander Broomfield,James Davison,Si Houn Hahn,Satoko Kumada,François Labarthe,Hirotaka Ohki,Samia Pichard,S Grace Prakalapakorn,Kristina An Haack,Barbara Kittner,Xianzhang Meng,Susan Sparks,Catherine Wilson,Atef Zaher,Yin-Hsiu Chien

Genetics in medicine : official journal of the American College of Medical Genetics（2023）

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摘要

These data support the positive clinical effect of avalglucosidase alfa in patients with infantile-onset Pompe disease previously declining on alglucosidase alfa.

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关键词

Acid α-glucosidase (GAA) deficiency,Alglucosidase alfa,Avalglucosidase alfa,Enzyme replacement therapy,Infantile-onset Pompe disease

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