European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy-Reference-Cited by-同舟云学术

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

Published:2020-09 Issue: Volume:28 Page:38-43
ISSN:1090-3798
Container-title:European Journal of Paediatric Neurology
language:en
Short-container-title:European Journal of Paediatric Neurology

Author:

Kirschner Janbernd^ORCID,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,Muntoni Francesco

Funder

Biogen

AveXis

Ionis

Roche

Publisher

Elsevier BV

Subject

Clinical Neurology,General Medicine,Pediatrics, Perinatology, and Child Health

Reference35 articles.

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2. Correlation between SMA type and SMN2 copy number revisited: an analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases;Calucho;Neuromuscul. Disord.,2018

3. Advances in treatment of spinal muscular atrophy - new phenotypes, new challenges, new implications for care;Schorling;J. Neuromuscul. Dis.,2020

4. Single-dose gene-replacement therapy for spinal muscular atrophy;Mendell;N. Engl. J. Med.,2017

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