Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial-Reference-Cited by-同舟云学术

Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial

Published:2024-02 Issue:2 Volume:141 Page:108121
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Toscano Antonio,Pollissard Laurence,Msihid Jérôme,van der Beek Nadine,Kishnani Priya S.,Dimachkie Mazen M.,Berger Kenneth I.,DasMahapatra Pronabesh,Thibault Nathan,Hamed Alaa,Zhou Tianyue,Haack Kristina An,Schoser Benedikt

Funder

Sanofi

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference37 articles.

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3. Multisystem late onset Pompe disease (LOPD): an update on clinical aspects;Toscano;Ann. Translat. Med.,2019

4. Insight into the phenotype of infants with Pompe disease identified by newborn screening with the common c.-32-13T>G "late-onset";Rairikar;GAA Variant Mol. Genet. Metab.,2017

5. Early-onset of symptoms and clinical course of Pompe disease associated with the c.-32–13 T > G variant;Herbert;Mol. Genet. Metab.,2019

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