Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study

Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study

Abstract Background Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterized by degeneration of the anterior horn cells of the spinal cord. Nusinersen has been covered by public healthcare in France since May 2017. The aim of this article is to report results afte...

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Bibliographic Details
Main Authors: Frédérique Audic, Marta Gomez Garcia de la Banda, Delphine Bernoux, Paola Ramirez-Garcia, Julien Durigneux, Christine Barnerias, Arnaud Isapof, Jean-Marie Cuisset, Claude Cances, Christian Richelme, Carole Vuillerot, Vincent Laugel, Juliette Ropars, Cécilia Altuzarra, Caroline Espil-Taris, Ulrike Walther-Louvier, Pascal Sabouraud, Mondher Chouchane, Catherine Vanhulle, Valérie Trommsdorff, Anne Pervillé, Hervé Testard, Emmanuelle Lagrue, Catherine Sarret, Anne-Laude Avice, Pierre Beze-Beyrie, Vanessa Pauly, Susana Quijano-Roy, Brigitte Chabrol, Isabelle Desguerre
Format: Article
Language:English
Published: BMC 2020-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Spinal muscular atrophy type I
Spinal muscular atrophy type II
Nusinersen
Motor function measure
MFM
Online Access:http://link.springer.com/article/10.1186/s13023-020-01414-8

Internet

http://link.springer.com/article/10.1186/s13023-020-01414-8

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