Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French rea...
Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
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Author / Creator
Audic, Frédérique , de la Banda, Marta Gomez Garcia , 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 and Desguerre, Isabelle
Publisher
London: BioMed Central Ltd
Journal title
Language
English
Formats
Publication information
Publisher
London: BioMed Central Ltd
Subjects
More information
Scope and Contents
Contents
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 after 1 year of treatment with intrathecal nusinersen in children with SMA...
Alternative Titles
Full title
Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
Authors, Artists and Contributors
Author / Creator
de la Banda, Marta Gomez Garcia
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
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_318647e3ed8f4622944ee433cb371756
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_318647e3ed8f4622944ee433cb371756
Other Identifiers
ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-020-01414-8
