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

In this comparative effectiveness study of children with SMA1, gene therapy was associated with lower incidence of UCR and fewer supportive care needs vs nusinersen. These exploratory findings warrant confirmation in larger studies.

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Publication Details

Journal
JAMA Network Open
Published
2025-10-08
DOI
https://doi.org/10.1001/jamanetworkopen.2025.36348
Citations
1
Primary Topic
Neurogenetic and Muscular Disorders Research
Type
article
Field-Weighted Citation Impact
1.63
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Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1

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

Edoardo Malfatti, Philippe Merle, Cyril Schweitzer, Jean‐Baptiste Davion, Anne-Laure Kaminsky, Klaus Dieterich, Marco Spinazzi, Christian Richelme, Lionelle Nkam, Tanya Stojkovic, C. Cancès, Susana Quijano‐Roy, Mélanie Fradin, Isabelle Desguerre, Elisabeth Sarrazin, Narcisse Elenga, Guilhem Solé, Vincent Laugel, J. Durigneux, Marine Guichard, Marta Gómez‐García de la Banda, Andoni Echaniz‐Laguna, Lamiae Grimaldi‐Bensouda, Olivier Flabeau, Maud Michaud, B. Chabrol, Caroline Espil‐Taris, Gaëlle Gousse, Yann Péréon, F. Boyer, Rocio García-Uzquiano, Laure Le Goff, Mondher Chouchane, Sylvain Nollet, Pascal Sabouraud, Christine Barnérias, Elisa De La Cruz, Frédérique Audic, Ulrike Walther‐Louvier, Patrick C. Berquin, Carole Vuillerot, Rachel Froget, Catherine Sarret, Sabrina Sacconi, Djillali Annane, Marie Thibaud, Caroline Espil, Aleksandra Nadaj‐Pakleza, Karima Ghorab, Catherine Vanhulle, P. Bèze-Beyrie, Sandra Mercier, Anne Pervillé, Shahram Attarian, Pascal Cintas, Sophie Duclos, Juliette Ropars, Nathalie Bach, Agnès Jacquin-Piques, Kumaran Deiva, Lucie Guyant‐Maréchal, Leïla Lazaro, C. Lambert, Arnaud Isapof, Rémi Bellance, C. Halbert, Jon Andoni Urtizberea, Pascal Laforêt, Jean‐Baptiste Noury, Marie-Christine Nouges, Valérie Trommsdorf
article en
1 citations

Abstract

In this comparative effectiveness study of children with SMA1, gene therapy was associated with lower incidence of UCR and fewer supportive care needs vs nusinersen. These exploratory findings warrant confirmation in larger studies.

JAMA Network OpenVol. 8(10)
Université Claude Bernard Lyon 1 (FR), Hôpital Necker-Enfants Malades (FR), Université de Bordeaux (FR), Inserm (FR), Université Fédérale de Toulouse Midi-Pyrénées (FR), Université de Versailles Saint-Quentin-en-Yvelines (FR), Université de Montpellier (FR), Université de Lille (FR), Université Paris-Saclay (FR), Sorbonne Université (FR), Hôpital Femme Mère Enfant (FR), Hôpital Armand-Trousseau (FR), Assistance Publique Hôpitaux de Marseille (FR), Center for the Study of Social Policy (US), Centre Hospitalier Universitaire de Montpellier (FR), Assistance Publique – Hôpitaux de Paris (FR), Hospices Civils de Lyon (FR), Institut NeuroMyoGène (FR), Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie appliquées (FR), Centre Hospitalier Régional Universitaire de Brest (FR), Hôpital Raymond-Poincaré (FR), Laboratoire de Traitement de l'Information Médicale (FR), Hôpital de la Timone (FR), Université d'Angers (FR), Université de Strasbourg (FR)
Openalex Percentile: Top 13%
Neurogenetic and Muscular Disorders Research
1.63
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