Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study | Orphanet Journal of Rare Diseases | Full Text

Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study | Orphanet Journal of Rare Diseases | Full Text

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

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 F...

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…

Citation:Orphanet Journal of Rare Diseases 2020 15:148

Content type:Research

 

Published on: 12 June 2020

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