Three years pilot of spinal muscular atrophy newborn screening turned into official program in Southern Belgium

Three new therapies for spinal muscular atrophy (SMA) have been approved by the United States Food and Drug Administration and the European Medicines Agency since 2016. Although these new therapies improve the quality of life of patients who are symptomatic at first treatment, administration before...

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Detalles Bibliográficos
Autores principales: Boemer, François, Caberg, Jean-Hubert, Beckers, Pablo, Dideberg, Vinciane, di Fiore, Samantha, Bours, Vincent, Marie, Sandrine, Dewulf, Joseph, Marcelis, Lionel, Deconinck, Nicolas, Daron, Aurore, Blasco-Perez, Laura, Tizzano, Eduardo, Hiligsmann, Mickaël, Lombet, Jacques, Pereira, Tatiana, Lopez-Granados, Lucia, Shalchian-Tehran, Sarvnaz, van Assche, Véronique, Willems, Arabelle, Huybrechts, Sofie, Mast, Bénédicte, van Olden, Rudolf, Dangouloff, Tamara, Servais, Laurent
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8497564/
https://www.ncbi.nlm.nih.gov/pubmed/34620959
http://dx.doi.org/10.1038/s41598-021-99496-2

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