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Authors
Schoser, Benedikt https://orcid.org/0000-0002-2757-8131
Domínguez-González, Cristina https://orcid.org/0000-0001-5151-988X
Laforet, Pascal https://orcid.org/0000-0001-8509-9107
Hahn, Andreas https://orcid.org/0000-0003-0235-5211
Gissen, Paul https://orcid.org/0000-0002-9712-6122
Kostera-Pruszczyk, Anna https://orcid.org/0000-0002-7309-1914
Thalmeier, Andreas https://orcid.org/0009-0009-5455-5603
Vissing, John https://orcid.org/0000-0001-6144-8544
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Funding
Funding for this research was provided by:
Astellas Gene Therapies
Klinikum der Universität München
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Article History
Received: 17 November 2025
Revised: 3 February 2026
Accepted: 5 February 2026
First Online: 14 February 2026
Declarations
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: Benedikt Schoser has received research grants from the EU Horizon 2022 programs ComPaSS, PaLaDIn, and ENTRY-DM, PepGen DM research; received consulting fees from Alexion, Amicus, Astellas, Avidity, Denali, Dyne, Sanofi, and Johnson&Johnson; honoraria from Alexion, Argenx, Astellas, Dyne, Kedrion, and Sanofi; support for attending meetings and/or travel from Astellas, Amicus, and Sanofi; have participated in advisory boards for Amicus, Astellas, Avidity, Dyne, and Encoded; and declares no stocks or shares. Cristina Dominguez has received research grants from UCB Pharma, Pretzel Therapeutics and Instituto de Salud Carlos III; received speaker's honoraria or consulting fees from Astellas, Amicus, Pretzel Therapeutics, Sanofi, Roche, and UCB; and declares no stocks or shares. Pascal Laforet has received research grants from Amicus Therapeutics and Sanofi; consulting fees or speaker's honoraria or support for attending meetings and/or travel from Amicus Therapeutics, Astellas, and Sanofi; and declares no stocks or shares. Paul Gissen has received consulting fees from BioMarin and Travere Therapeutics; received speaker's honoraria from BioMarin; is a member of the MRC Molecular and Cellular Medicine Board, the Scientific Committee for International Niemann Pick Disease Registry, the Medical and Scientific Advisory Board for Batten Disease Family Association, and of Telethon Grants; and declares shares of Bloomsbury Genetic Therapies. Anna Kostera-Pruszczyk has received research grants from Sanofi, Biogen, and Kedrion; speaker's honoraria from Novartis, Roche, Biogen, Alexion, UCB, Takeda, CSL Behring, and AstraZeneca; is a member of the Council of Rare Diseases (Advisory to Ministry of Health); and declares no stocks or shares. Andreas Thalmeier has received consulting fees from Astellas and Roche. John Vissing has received research grants from NOVO Nordic foundation and an EU grant for Becker muscular dystrophy; consulting fees from Sanofi, Dyne Therapeutics, and Roche; speaker's honoraria from Edgewise Therapeutics, UCB Biopharma, Alexion Pharmaceuticals, and Janssen Pharmaceuticals; has participated in advisory boards for Dyne Therapeutics; and declares no stocks or shares. Andreas Hahn has received research grants from Sanofi and consulting and/or speaker’s honoraria from Amicus, Sanofi, Astellas, Pfizer, Roche, and Novartis.
: The manuscript does not contain clinical studies or patient data.
