NEO1/NEO-EXT Studies: Long-term Muscle Quantitative Magnetic Resonance Imaging and Functional Efficacy in Adults With Late-onset Pompe Disease (LOPD) on Avalglucosidase Alfa Treatment (P8-4.010)

Shafeeq Ladha; Pierre G. Carlier; John Vissing; Mazen M. Dimachkie; Barry J. Byrne; Richard J. Barohn; Priya S. Kishnani; Eugen Mengel; Sabrina Sacconi; Jaya Trivedi; Peter Young; Kristina An Haack; Nicole M. Armstrong; P. Miossec; Nathan Thibault; Susan Sparks; Benedikt Schoser; Jordi Díaz‐Manera

doi:10.1212/wnl.0000000000206122

Abstract

To assess changes in muscle fat fraction (FF) and functional efficacy in patients with late-onset Pompe disease (LOPD) after long-term treatment with avalglucosidase alfa (AVA).

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Ladha et al. (2024) studied this question.

synapsesocial.com/papers/68e6fdadb6db643587677d74 https://doi.org/10.1212/wnl.0000000000206122

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NEO1/NEO-EXT Studies: Long-term Muscle Quantitative Magnetic Resonance Imaging and Functional Efficacy in Adults With Late-onset Pompe Disease (LOPD) on Avalglucosidase Alfa Treatment (P8-4.010)

Abstract

Discussion

Cite This Study

NEO1/NEO-EXT Studies: Long-term Muscle Quantitative Magnetic Resonance Imaging and Functional Efficacy in Adults With Late-onset Pompe Disease (LOPD) on Avalglucosidase Alfa Treatment (P8-4.010)

Abstract

Discussion

Cite This Study

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