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

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

Abstract 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, administratio...

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Auteurs principaux: François Boemer, Jean-Hubert Caberg, Pablo Beckers, Vinciane Dideberg, Samantha di Fiore, Vincent Bours, Sandrine Marie, Joseph Dewulf, Lionel Marcelis, Nicolas Deconinck, Aurore Daron, Laura Blasco-Perez, Eduardo Tizzano, Mickaël Hiligsmann, Jacques Lombet, Tatiana Pereira, Lucia Lopez-Granados, Sarvnaz Shalchian-Tehran, Véronique van Assche, Arabelle Willems, Sofie Huybrechts, Bénédicte Mast, Rudolf van Olden, Tamara Dangouloff, Laurent Servais
Format: article
Langue:EN
Publié: Nature Portfolio 2021
Sujets:
Medicine
R
Science
Q
Accès en ligne:https://doaj.org/article/aee7b58b35494c7a81456a3ae885e5c8
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https://doaj.org/article/aee7b58b35494c7a81456a3ae885e5c8

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