Article
Three years pilot of spinal muscular atrophy newborn screening turned into official program in Southern Belgium.
Scientific reports - 7 Oct 2021
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
Abstract excerpt
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 the onset of symptoms is significantly more effective. As a consequence, newborn screening programs have been...
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