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

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 Eur...

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Detalles Bibliográficos
Autores principales:	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, Muntoni, Francesco
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	European Paediatric Neurology Society. Published by Elsevier Ltd. 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7347351/ https://www.ncbi.nlm.nih.gov/pubmed/32763124 http://dx.doi.org/10.1016/j.ejpn.2020.07.001

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