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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

Document Type

Article

Author

Kishnani, P.S.; Kronn, D.; Brassier, A.; Pichard, S.; Broomfield, A.; Davison, J.; Hahn, S.H.; Kumada, S.; Labarthe, F.; Ohki, H.; Prakalapakorn, S.G.; Haack, K.A.; Kittner, B.; Meng, X.; Sparks, S.; Wilson, C.; Zaher, A.; Chien, Y.-H.

Source

In: Genetics in Medicine. (Genetics in Medicine, February 2023, 25(2))

Subject

Language

English

ISSN

15300366
10983600

Online Access

Full Text (ScienceDirect) Full Text (ScienceDirect 종량제) Full Text (ScienceDirect O/A) Web of Science JCR 저널정보 Scopus Find it@PNU

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