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

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

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DOI: 10.6084/m9.figshare.c.8819380.v1
Published: 2026
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Publisher: figshare
Open access: yes

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Cite this work

BibTeX

@article{allodium:10.6084/m9.figshare.c.8819380.v1,
  title = {Use of European registries to characterise the changing landscape of disease progression and treatment of spinal muscular atrophy (SMA): opportunities, pitfalls and challenges},
  author = {Nicolas Deltour and Seung Yeon Lee and Elizabeth M. Garry and Liza R. Gibbs and Shane McElwee and Renee M. Sajedian and Emmanuelle Jacquot and Siyana Kurteva and Anabel Ferreras and Annie Poll and Ben Porter and Rhian Davies and Jack Kennedy and Maggie C. Walter and Simone Thiele and Marlène Jagut and Marjan Cosyns and Liesbeth de Waele and Nicolas Deconinck and Jana Haberlová and Lenka Mokrá and Maria Grazia Cattinari and Eduardo F. Tizzano and Chiara Marini-Bettolo and Lindsay Murphy and Anne-Berit Ekström and Miriam Rodrigues and Anna Ambrosini and Victoria Hodgkinson and Michela Guglieri and Mencia de Lemus Belmonte and Kieran C. Breen and Laurent Servais},
  year = {2026},
  doi = {10.6084/m9.figshare.c.8819380.v1},
  url = {https://doi.org/10.6084/m9.figshare.c.8819380.v1}
}

RIS

TY  - JOUR
TI  - Use of European registries to characterise the changing landscape of disease progression and treatment of spinal muscular atrophy (SMA): opportunities, pitfalls and challenges
AU  - Nicolas Deltour
AU  - Seung Yeon Lee
AU  - Elizabeth M. Garry
AU  - Liza R. Gibbs
AU  - Shane McElwee
AU  - Renee M. Sajedian
AU  - Emmanuelle Jacquot
AU  - Siyana Kurteva
AU  - Anabel Ferreras
AU  - Annie Poll
AU  - Ben Porter
AU  - Rhian Davies
AU  - Jack Kennedy
AU  - Maggie C. Walter
AU  - Simone Thiele
AU  - Marlène Jagut
AU  - Marjan Cosyns
AU  - Liesbeth de Waele
AU  - Nicolas Deconinck
AU  - Jana Haberlová
AU  - Lenka Mokrá
AU  - Maria Grazia Cattinari
AU  - Eduardo F. Tizzano
AU  - Chiara Marini-Bettolo
AU  - Lindsay Murphy
AU  - Anne-Berit Ekström
AU  - Miriam Rodrigues
AU  - Anna Ambrosini
AU  - Victoria Hodgkinson
AU  - Michela Guglieri
AU  - Mencia de Lemus Belmonte
AU  - Kieran C. Breen
AU  - Laurent Servais
PY  - 2026
DO  - 10.6084/m9.figshare.c.8819380.v1
UR  - https://doi.org/10.6084/m9.figshare.c.8819380.v1
ER  -

APA

Deltour, N., Lee, S. Y., Garry, E. M., Gibbs, L. R., McElwee, S., Sajedian, R. M., Jacquot, E., Kurteva, S., Ferreras, A., Poll, A., Porter, B., Davies, R., Kennedy, J., Walter, M. C., Thiele, S., Jagut, M., Cosyns, M., Waele, L. D., Deconinck, N., Haberlová, J., Mokrá, L., Cattinari, M. G., Tizzano, E. F., Marini-Bettolo, C., Murphy, L., Ekström, A., Rodrigues, M., Ambrosini, A., Hodgkinson, V., Guglieri, M., Belmonte, M. D. L., Breen, K. C., & Servais, L. (2026). Use of European registries to characterise the changing landscape of disease progression and treatment of spinal muscular atrophy (SMA): opportunities, pitfalls and challenges. https://doi.org/10.6084/m9.figshare.c.8819380.v1

Source records

datacite · retrieved 2026-11-10T12:35:42.090Z