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: | , , , , , , , , , , , , , , , , , , , , , , , , |
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| Format: | article |
| Langue: | EN |
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Nature Portfolio
2021
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| Accès en ligne: | https://doaj.org/article/aee7b58b35494c7a81456a3ae885e5c8 |
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