Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1

Juliette Ropars, Claude Cances, Rocio Garcia-Uzquiano, Marta Gomez-Garcia de la Banda, Christine Barnerias, Frédérique Audic, Julien Durigneux, Cécile Halbert, Lionelle Nkam, Vincent Laugel, Caroline Espil, Ulrike Walther-Louvier, Jean-Baptiste Davion, Arnaud Isapof, Laure Le Goff, Isabelle Desguerre, Susana Quijano-Roy, Lamiae Grimaldi, Unknown, Djillali Annane, Shahram Attarian, Nathalie Bach, Rémi Bellance, Patrick Berquin, Pierre Beze-Beyrie, Francois Constant Boyer, Brigitte Chabrol, Mondher Chouchane, Ariane Choumert, Pascal Cintas, Elisa De La Cruz, Kumaran Deiva, Klaus Dieterich, Sophie Duclos, Andoni Echaniz-Laguna, Narcisse Elenga, Caroline Espil-Taris, Olivier Flabeau, Melanie Fradin, Rachel Froget, Karima Ghorab, Gaëlle Gousse, Marine Guichard, Lucie Guyant-Marechal, Agnès Jacquin-Piques, Anne-Laure Kaminsky, Pascal Laforet, Clémentine Lambert, Leila Lazaro, Edoardo Malfatti, Sandra Mercier, Philippe Edouard Merle, Maud Michaud, Aleksandra Nadaj-Pakleza, Sylvain Nollet, Marie-Christine Nouges, Jean-Baptiste NOURY, Yann Pereon, Anne Pervillé, Christian Richelme, Pascal Sabouraud, Sabrina Sacconi, Elisabeth Sarrazin, Catherine Sarret, Cyril Schweitzer, Guilhem Sole, Marco Spinazzi, Tanya Stojkovic, Marie Thibaud, Valérie Trommsdorf, Jon Andoni Urtizberea, Catherine Vanhulle, Carole Vuillerot

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DOI: 10.1001/jamanetworkopen.2025.36348
Published: 2025-10-08
Container: JAMA Network Open
Publisher: American Medical Association (AMA)
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Cite this work

BibTeX

@article{allodium:10.1001/jamanetworkopen.2025.36348,
  title = {Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1},
  author = {Juliette Ropars and Claude Cances and Rocio Garcia-Uzquiano and Marta Gomez-Garcia de la Banda and Christine Barnerias and Frédérique Audic and Julien Durigneux and Cécile Halbert and Lionelle Nkam and Vincent Laugel and Caroline Espil and Ulrike Walther-Louvier and Jean-Baptiste Davion and Arnaud Isapof and Laure Le Goff and Isabelle Desguerre and Susana Quijano-Roy and Lamiae Grimaldi and Unknown and Djillali Annane and Shahram Attarian and Nathalie Bach and Rémi Bellance and Patrick Berquin and Pierre Beze-Beyrie and Francois Constant Boyer and Brigitte Chabrol and Mondher Chouchane and Ariane Choumert and Pascal Cintas and Elisa De La Cruz and Kumaran Deiva and Klaus Dieterich and Sophie Duclos and Andoni Echaniz-Laguna and Narcisse Elenga and Caroline Espil-Taris and Olivier Flabeau and Melanie Fradin and Rachel Froget and Karima Ghorab and Gaëlle Gousse and Marine Guichard and Lucie Guyant-Marechal and Agnès Jacquin-Piques and Anne-Laure Kaminsky and Pascal Laforet and Clémentine Lambert and Leila Lazaro and Edoardo Malfatti and Sandra Mercier and Philippe Edouard Merle and Maud Michaud and Aleksandra Nadaj-Pakleza and Sylvain Nollet and Marie-Christine Nouges and Jean-Baptiste NOURY and Yann Pereon and Anne Pervillé and Christian Richelme and Pascal Sabouraud and Sabrina Sacconi and Elisabeth Sarrazin and Catherine Sarret and Cyril Schweitzer and Guilhem Sole and Marco Spinazzi and Tanya Stojkovic and Marie Thibaud and Valérie Trommsdorf and Jon Andoni Urtizberea and Catherine Vanhulle and Carole Vuillerot},
  year = {2025},
  journal = {JAMA Network Open},
  doi = {10.1001/jamanetworkopen.2025.36348},
  url = {https://doi.org/10.1001/jamanetworkopen.2025.36348}
}

RIS

TY  - JOUR
TI  - Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1
AU  - Juliette Ropars
AU  - Claude Cances
AU  - Rocio Garcia-Uzquiano
AU  - Marta Gomez-Garcia de la Banda
AU  - Christine Barnerias
AU  - Frédérique Audic
AU  - Julien Durigneux
AU  - Cécile Halbert
AU  - Lionelle Nkam
AU  - Vincent Laugel
AU  - Caroline Espil
AU  - Ulrike Walther-Louvier
AU  - Jean-Baptiste Davion
AU  - Arnaud Isapof
AU  - Laure Le Goff
AU  - Isabelle Desguerre
AU  - Susana Quijano-Roy
AU  - Lamiae Grimaldi
AU  - Unknown
AU  - Djillali Annane
AU  - Shahram Attarian
AU  - Nathalie Bach
AU  - Rémi Bellance
AU  - Patrick Berquin
AU  - Pierre Beze-Beyrie
AU  - Francois Constant Boyer
AU  - Brigitte Chabrol
AU  - Mondher Chouchane
AU  - Ariane Choumert
AU  - Pascal Cintas
AU  - Elisa De La Cruz
AU  - Kumaran Deiva
AU  - Klaus Dieterich
AU  - Sophie Duclos
AU  - Andoni Echaniz-Laguna
AU  - Narcisse Elenga
AU  - Caroline Espil-Taris
AU  - Olivier Flabeau
AU  - Melanie Fradin
AU  - Rachel Froget
AU  - Karima Ghorab
AU  - Gaëlle Gousse
AU  - Marine Guichard
AU  - Lucie Guyant-Marechal
AU  - Agnès Jacquin-Piques
AU  - Anne-Laure Kaminsky
AU  - Pascal Laforet
AU  - Clémentine Lambert
AU  - Leila Lazaro
AU  - Edoardo Malfatti
AU  - Sandra Mercier
AU  - Philippe Edouard Merle
AU  - Maud Michaud
AU  - Aleksandra Nadaj-Pakleza
AU  - Sylvain Nollet
AU  - Marie-Christine Nouges
AU  - Jean-Baptiste NOURY
AU  - Yann Pereon
AU  - Anne Pervillé
AU  - Christian Richelme
AU  - Pascal Sabouraud
AU  - Sabrina Sacconi
AU  - Elisabeth Sarrazin
AU  - Catherine Sarret
AU  - Cyril Schweitzer
AU  - Guilhem Sole
AU  - Marco Spinazzi
AU  - Tanya Stojkovic
AU  - Marie Thibaud
AU  - Valérie Trommsdorf
AU  - Jon Andoni Urtizberea
AU  - Catherine Vanhulle
AU  - Carole Vuillerot
PY  - 2025
JO  - JAMA Network Open
DO  - 10.1001/jamanetworkopen.2025.36348
UR  - https://doi.org/10.1001/jamanetworkopen.2025.36348
ER  -

APA

Ropars, J., Cances, C., Garcia-Uzquiano, R., Banda, M. G. D. L., Barnerias, C., Audic, F., Durigneux, J., Halbert, C., Nkam, L., Laugel, V., Espil, C., Walther-Louvier, U., Davion, J., Isapof, A., Goff, L. L., Desguerre, I., Quijano-Roy, S., Grimaldi, L., Unknown, Annane, D., Attarian, S., Bach, N., Bellance, R., Berquin, P., Beze-Beyrie, P., Boyer, F. C., Chabrol, B., Chouchane, M., Choumert, A., Cintas, P., Cruz, E. D. L., Deiva, K., Dieterich, K., Duclos, S., Echaniz-Laguna, A., Elenga, N., Espil-Taris, C., Flabeau, O., Fradin, M., Froget, R., Ghorab, K., Gousse, G., Guichard, M., Guyant-Marechal, L., Jacquin-Piques, A., Kaminsky, A., Laforet, P., Lambert, C., Lazaro, L., Malfatti, E., Mercier, S., Merle, P. E., Michaud, M., Nadaj-Pakleza, A., Nollet, S., Nouges, M., NOURY, J., Pereon, Y., Pervillé, A., Richelme, C., Sabouraud, P., Sacconi, S., Sarrazin, E., Sarret, C., Schweitzer, C., Sole, G., Spinazzi, M., Stojkovic, T., Thibaud, M., Trommsdorf, V., Urtizberea, J. A., Vanhulle, C., & Vuillerot, C. (2025). Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1. JAMA Network Open. https://doi.org/10.1001/jamanetworkopen.2025.36348

Source records

crossref · retrieved 2026-09-27T10:59:58.059Z