Use of European registries to characterise the changing landscape of disease progression and treatment of spinal muscular atrophy (SMA): opportunities, pitfalls and challenges

Orphanet Journal of Rare Diseases · Published 2026-08-05 · DOI 10.1186/s13023-026-04491-3

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Authors (62)

Nicolas Deltour, Seung Yeon Lee, Elizabeth M. Garry, Liza R. Gibbs, Shane McElwee, Renee M. Sajedian, Emmanuelle Jacquot, Siyana Kurteva, Anabel Ferreras, Annie Poll, Ben Porter, Rhian Davies, Jack Kennedy, Maggie C. Walter, Simone Thiele, Marlène Jagut, Marjan Cosyns, Liesbeth de Waele, Nicolas Deconinck, Jana Haberlová, Lenka Mokrá, Maria Grazia Cattinari, Eduardo F. Tizzano, Chiara Marini-Bettolo, Lindsay Murphy, Anne-Berit Ekström, Miriam Rodrigues, Anna Ambrosini, Victoria Hodgkinson, Michela Guglieri, Mencia de Lemus Belmonte, Kieran C. Breen, Laurent Servais, The Study Investigators Group, Aurore Daron, Stephanie Delstanche, Laura Vanden Brande, Alain Maertens de Noordhout, Stéphanie Paquay, Van den Bergh, Vinciane Van Parys, Nicolas Dubuisson, Gauthier Remiche, Florence Christiaens, Jonathan Baets, Alicia Alonso-Jimenez, Diane Beysen, Véronique Bissay, Nathalie Smeets, Karine Pelc, Katrien De Braekeleer, Jan De Bleecker, Arnaud Vanlander, Sarah Herdewyn, Kristl Claeys, Geertrui Peirens, Philip Van Damme, Anna-Karin Kroksmark, Olesja Parmová, Lenka Juříková, Kryštof Prášil, Neil Bennett

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Publication details

Year
2026

Citation

Deltour, N., Lee, S., Garry, E., et al. (2026). Use of European registries to characterise the changing landscape of disease progression and treatment of spinal muscular atrophy (SMA): opportunities, pitfalls and challenges. Orphanet Journal of Rare Diseases. https://doi.org/10.1186/s13023-026-04491-3

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