Article (Scientific journals)
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.
Kirschner, Janbernd; Butoianu, Nina; Goemans, Nathalie et al.
2020In European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society, 28, p. 38-43
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Keywords :
Biological Products/therapeutic use; Consensus; Genetic Therapy/methods; Humans; Infant; Muscular Atrophy, Spinal/genetics/therapy; Patient Selection; Recombinant Fusion Proteins/therapeutic use; Gene therapy; Nusinersen; Onasemnogene abeparvovec; SMN1; SMN2; Spinal muscular atrophy; Zolgensma
Abstract :
[en] Spinal muscular atrophy (SMA) used to be one of the most common genetic causes of infant mortality. New disease modifying treatments have changed the disease trajectories and most impressive results are seen if treatment is initiated in the presymptomatic phase of the disease. Very recently, the European Medicine Agency approved Onasemnogene abeparvovec (Zolgensma®) for the treatment of patients with SMA with up to three copies of the SMN2 gene or the clinical presentation of SMA type 1. While this broad indication provides new opportunities, it also triggers discussions on the appropriate selection of patients in the context of limited available evidence. To aid the rational use of Onasemnogene abeparvovec for the treatment of SMA, a group of European neuromuscular experts presents in this paper eleven consensus statements covering qualification, patient selection, safety considerations and long-term monitoring.
Disciplines :
Neurology
Pediatrics
Author, co-author :
Kirschner, Janbernd
Butoianu, Nina
Goemans, Nathalie
Haberlova, Jana
Kostera-Pruszczyk, Anna
Mercuri, Eugenio
van der Pol, W. Ludo
Quijano-Roy, Susana
Sejersen, Thomas
Tizzano, Eduardo F.
Ziegler, Andreas
Servais, Laurent ;  Université de Liège - ULiège > Département des sciences cliniques > Département des sciences cliniques
Muntoni, Francesco
More authors (3 more) Less
Language :
English
Title :
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.
Publication date :
September 2020
Journal title :
European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
ISSN :
1090-3798
eISSN :
1532-2130
Volume :
28
Pages :
38-43
Peer reviewed :
Peer reviewed
Commentary :
Copyright © 2020 European Paediatric Neurology Society. Published by Elsevier Ltd. All rights reserved. DOI: 10.1016/j.ejpn.2020.07.001
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