Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials

Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials

Background: The efficacy of avalglucosidase alfa (AVA) versus alglucosidase alfa (ALG) on forced vital capacity percent predicted (FVCpp) in patients with late-onset Pompe disease (LOPD) has been assessed in the Phase 3 COMET trial (NCT02782741). Due to the rarity of LOPD and thus small sample size...

Full description

Bibliographic Details
Main Authors: Tahseen Mozaffar, Lionel Riou França, Jérôme Msihid, Pragya Shukla, Irina Proskorovsky, Tianyue Zhou, Magali Periquet, Kristina An Haack, Laurence Pollissard, Volker Straub
Format: Article
Language:English
Published: Elsevier 2024-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Late-onset Pompe disease
Enzyme replacement therapy
Avalglucosidase alfa
Alglucosidase alfa
Respiratory
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426924000624

Internet

http://www.sciencedirect.com/science/article/pii/S2214426924000624

Similar Items