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2025
Mark Gene therapy in advanced metachromatic leukodystrophy : tempering expectations
Schoenmakers, Daphne H. ; Beerepoot, Shanice ; Adang, Laura A. ; Asbreuk, Marije A.B.C. ; Bergner, Caroline G. ; Bley, Annette E. ; Boelens, Jaap Jan ; Calbi, Valeria ; Darling, Alejandra and Eklund, Erik ^LU , et al. (2025) In Protein and Cell 16(1). p.12-15
- Contribution to journal › Debate/Note/Editorial
Mark Metachromatic Leukodystrophy : New Therapy Advancements and Emerging Research Directions
Asbreuk, Marije A B C ; Schoenmakers, Daphne H ; Adang, Laura Ann ; Beerepoot, Shanice ; Bergner, Caroline ; Bley, Annette ; Boelens, Jaap Jan ; Bugiani, Marianna ; Calbi, Valeria and García-Cazorla, Àngeles , et al. (2025) In Neurology 105(2).
- Contribution to journal › Scientific review
Mark Key lessons from the first international treatment eligibility committee : the case of metachromatic leukodystrophy
Schoenmakers, Daphne H ; Asbreuk, Marije A B C ; Martin, Tamara ; Datema, Mareen ; Beerepoot, Shanice ; Inbar-Feigenberg, Michal ; Groeschel, Samuel ; Kehrer, Christiane ; Øberg, Andreas and Sevin, Caroline , et al. (2025) In European Journal of Paediatric Neurology 57. p.72-81
- Contribution to journal › Article
2024
Mark Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries
Schoenmakers, Daphne H. ; Mochel, Fanny ; Adang, Laura A. ; Boelens, Jaap Jan ; Calbi, Valeria ; Eklund, Erik A. ^LU ; Grønborg, Sabine W. ; Fumagalli, Francesca ; Groeschel, Samuel and Lindemans, Caroline , et al. (2024) In Orphanet Journal of Rare Diseases 19(1).
- Contribution to journal › Article
Mark Newborn screening in metachromatic leukodystrophy – European consensus-based recommendations on clinical management
Laugwitz, Lucia ; Schoenmakers, Daphne H. ; Adang, Laura A. ; Beck-Woedl, Stefanie ; Bergner, Caroline ; Bernard, Geneviève ; Bley, Annette ; Boyer, Audrey ; Calbi, Valeria and Dekker, Hanka , et al. (2024) In European Journal of Paediatric Neurology 49. p.141-154
- Contribution to journal › Article

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