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

Boemer, François; Caberg, Jean‐Hubert; Beckers, Pablo; Dideberg, Vinciane; di Fiore, Samantha; Bours, Vincent; Marie, Sandrine; P. Dewulf, Joseph; Marcélis, Lionel; Deconinck, Nicolas; Daron, Aurore; Campello Blasco, Laura; F. 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

doi:10.1038/s41598-021-99496-2

Public

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

Published in Scientific Reports • Oct 7, 2021

Authors:

François Boemer

Jean‐Hubert Caberg

Pablo Beckers

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, administration before ...

Subject

SMN1

Spinal muscular atrophy

SMA*

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