First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease

This real-world study suggests that transitions between ERT preparations are generally feasible and associated with clinical stability in LOPD. Switching may represent a useful strategy in patients, particularly when efficacy concerns arise. Standardized prospective studies with systematic monitoring of immunogenicity and efficacy according to the 2024 EOPC guideline are recommended to confirm these findings.

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Publication Details

Journal
Journal of Neurology
Published
2026-02-01
DOI
https://doi.org/10.1007/s00415-026-13689-1
Primary Topic
Lysosomal Storage Disorders Research
Type
article
Field-Weighted Citation Impact
0.00
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article

First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease

A Rosenbohm, Stephan Wenninger, Karl Christian Knop, Natalia García-Angarita et al.
Journal of Neurology
Lysosomal Storage Disorders Research
article

First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease

A Rosenbohm, Stephan Wenninger, Karl Christian Knop, Natalia García-Angarita, A. Güttsches, N. Nguyen-Younossi, Tanja Fangerau, Daniel H. Mendelsohn, Guljan Shahyrova, Cornelia Kornblum, Benedikt Schoser
article en

Abstract

This real-world study suggests that transitions between ERT preparations are generally feasible and associated with clinical stability in LOPD. Switching may represent a useful strategy in patients, particularly when efficacy concerns arise. Standardized prospective studies with systematic monitoring of immunogenicity and efficacy according to the 2024 EOPC guideline are recommended to confirm these findings.

Journal of NeurologyVol. 273(2)
Universität Ulm (DE), University Hospital Bonn (DE), Friedrich Baur Stiftung (DE), Hamburg Institut (Germany) (DE), BG University Hospital Bergmannsheil Bochum (DE), University and Rehabilitation Clinics Ulm (DE), University Hospital Ulm (DE), Ludwig-Maximilians-Universität München (DE)
Openalex Percentile: Top 54%
Lysosomal Storage Disorders Research
DOIOpen Access PDFOpenAlex
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