European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy
Janbernd Kirschner, Nina Butoianu, Nathalie Goemans, Jana Haberlová, Anna Kostera‐Pruszczyk, Eugenio Mercuri, W. Ludo van der Pol, Susana Quijano‐Roy, Thomas Sejersen, Eduardo F. Tizzano, Andreas Ziegler, Laurent Servais, Francesco Muntoni
Topics & Concepts
Spinal muscular atrophySMA*MedicineContext (archaeology)DiseasePhysical medicine and rehabilitationPresentation (obstetrics)Intensive care medicinePathologySurgeryBiologyComputer scienceAlgorithmPaleontologyNeurogenetic and Muscular Disorders ResearchRNA modifications and cancerCongenital Anomalies and Fetal Surgery