A roadmap for a patient-centred approach to Pompe disease management

Implementing gene therapy for Pompe disease requires a coordinated multidisciplinary effort to overcome gaps in knowledge, infrastructure, and patient management.

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Publication Details

Journal
Journal of Neurology
Published
2026-02-01
DOI
https://doi.org/10.1007/s00415-026-13687-3
Primary Topic
Lysosomal Storage Disorders Research
Type
article
Field-Weighted Citation Impact
0.00

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article

A roadmap for a patient-centred approach to Pompe disease management

P. Lafôret, Andreas Thalmeier, Cristina Domínguez-González, Paul Gissen et al.
Journal of Neurology
Lysosomal Storage Disorders Research
article

A roadmap for a patient-centred approach to Pompe disease management

P. Lafôret, Andreas Thalmeier, Cristina Domínguez-González, Paul Gissen, Anna Kostera-Pruszczyk, Andreas Hahn, John Vissing, Benedikt Schoser
article en

Abstract

Implementing gene therapy for Pompe disease requires a coordinated multidisciplinary effort to overcome gaps in knowledge, infrastructure, and patient management.

Journal of NeurologyVol. 273(2)
University of Copenhagen (DK), Justus-Liebig-Universität Gießen (DE), Medical University of Warsaw (PL), Copenhagen University Hospital (DK), Rigshospitalet (DK), LMU Klinikum (DE), National Institute for Health and Care Research (GB), Assistance Publique – Hôpitaux de Paris (FR), Centre for Biomedical Network Research on Rare Diseases (ES), Hôpital Raymond-Poincaré (FR), Ludwig-Maximilians-Universität München (DE)
Pfizer, AstraZeneca, Sanofi, Biogen, Alexion Pharmaceuticals, CSL Behring, Batten Disease Family Association, Amicus Therapeutics, Astellas Pharma, Novo Nordisk Fonden, Medical Research Council, Instituto de Salud Carlos III, Janssen Pharmaceuticals, UCB Pharma
Partnerships for the goals
Openalex Percentile: Top 53%
Lysosomal Storage Disorders Research
DOIOpen Access PDFOpenAlex
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A roadmap for a patient-centred approach to Pompe disease management — P. Lafôret, Andreas Thalmeier, et al. · Journal of Neurology (2026) | TGRS Research Map | TGRS