About: European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy

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An Entity of Type : schema:ScholarlyArticle, within Data Space : covidontheweb.inria.fr associated with source document(s)

Attributes	Values
type	Academic Article research paper schema:ScholarlyArticle
isDefinedBy	Covid-on-the-Web dataset
title	European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy
Creator	Muntoni, Francesco Kirschner, Janbernd Cz, Butoianu, Nina Goemans, Nathalie Haberlova, Jana Haberlova@fnmotol, Jana Kostera-Pruszczyk, Anna Ludo Van Der Pol, W Mercuri, Eugenio Quijano-Roy, Susana Sejersen, Thomas Servais, Laurent Tizzano, Eduardo Ziegler, Andreas
source	Elsevier; Medline; PMC
abstract	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.
has issue date	2020-07-09(xsd:dateTime)
bibo:doi	10.1016/j.ejpn.2020.07.001
bibo:pmid	32763124
has license	no-cc
sha1sum (hex)	297cb91f04c796165bd0219b99d9904088a5c739
schema:url	https://doi.org/10.1016/j.ejpn.2020.07.001
resource representing a document's title	European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy
has PubMed Central identifier	PMC7347351
has PubMed identifier	32763124
schema:publication	Eur J Paediatr Neurol
resource representing a document's body	covid:297cb91f04c796165bd0219b99d9904088a5c739#body_text
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