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

Audic, Frédérique; Gomez Garcia de la Banda, Marta; Bernoux, Delphine; Ramirez-Garcia, Paola; Durigneux, Julien; Barnerias, Christine; Isapof, Arnaud; Cuisset, Jean-Marie; Cances, Claude; Richelme, Christian; Vuillerot, Carole; Laugel, Vincent; Ropars, Juliette; Altuzarra, Cécilia; Espil-Taris, Caroline; Walther-Louvier, Ulrike; Sabouraud, Pascal; Chouchane, Mondher; Vanhulle, Catherine; Trommsdorff, Valérie; Pervillé, Anne; Testard, Hervé; Lagrue, Emmanuelle; Sarret, Catherine; Avice, Anne-Laude; Beze-Beyrie, Pierre; Pauly, Vanessa; Quijano-Roy, Susana; Chabrol, Brigitte; Desguerre, Isabelle

doi:10.1186/s13023-020-01414-8

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

Shared by NobleBlocks on Jun 12, 2020 • 12:00 AM UTC

Authors:

Frédérique Audic

Marta Gomez Garcia de la Banda

Delphine Bernoux

Abstract

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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