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Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline
Titel:
Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline
Auteur:
Kronn, David Davison, James Brassier, Anaïs Broomfield, Alexander Hahn, Si Houn Kumada, Satoko Labarthe, François Ohki, Hirotaka Prakalapakorn, S. Grace Haack, Kristina An Meng, Xianzhang Sparks, Susan Tammireddy, Swathi Wilson, Catherine Zaher, Atef Zhou, Tianyue Chien, Yin-Hsiu Kishnani, Priya S.
Verschenen in:
Molecular genetics and metabolism
Paginering:
Jaargang 135 () nr. 2 pagina's S68
Jaar:
2022
Inhoud:
Uitgever:
Published by Elsevier B.V.
Bronbestand:
Elektronische Wetenschappelijke Tijdschriften
Details van artikel 216 van 350 gevonden artikelen