Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spina...
Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment
About this item
Full title
Author / Creator
Desguerre, Isabelle , Barrois, Rémi , Audic, Frédérique , Barnerias, Christine , Chabrol, Brigitte , Davion, Jean Baptiste , Durigneux, Julien , Espil-Taris, Caroline , Gomez-Garcia de la Banda, Marta , Guichard, Marine , Isapof, Arnaud , Nougues, Marie Christine , Laugel, Vincent , Le Goff, Laure , Mercier, Sandra , Pervillé, Anne , Richelme, Christian , Thibaud, Marie , Sarret, Catherine , Schweitzer, Cyril , Testard, Hervé , Trommsdorff, Valérie , Vanhulle, Catherine , Walther-Louvier, Ulrike , Altuzarra, Cécilia , Chouchane, Mondher , Ropars, Juliette , Quijano-Roy, Susana and Cances, Claude
Publisher
England: BioMed Central Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: BioMed Central Ltd
Subjects
More information
Scope and Contents
Contents
Spinal muscular atrophy type 1 (SMA1) is the most severe and early form of SMA, a genetic disease with motor neuron degeneration. Onasemnogene abeparvovec gene transfer therapy (GT) has changed the natural history of SMA1, but real-world data are scarce.
A French national expert committee identified 95 newly diagnosed treatment-naive SMA1 patien...
Alternative Titles
Full title
Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment
Authors, Artists and Contributors
Author / Creator
Barrois, Rémi
Audic, Frédérique
Barnerias, Christine
Chabrol, Brigitte
Davion, Jean Baptiste
Durigneux, Julien
Espil-Taris, Caroline
Gomez-Garcia de la Banda, Marta
Guichard, Marine
Isapof, Arnaud
Nougues, Marie Christine
Laugel, Vincent
Le Goff, Laure
Mercier, Sandra
Pervillé, Anne
Richelme, Christian
Thibaud, Marie
Sarret, Catherine
Schweitzer, Cyril
Testard, Hervé
Trommsdorff, Valérie
Vanhulle, Catherine
Walther-Louvier, Ulrike
Altuzarra, Cécilia
Chouchane, Mondher
Ropars, Juliette
Quijano-Roy, Susana
Cances, Claude
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_15c4bb3f54a24e75bc25f097944bb5f3
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_15c4bb3f54a24e75bc25f097944bb5f3
Other Identifiers
ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-024-03326-3
