Nusinersen: Single-Centre Real-Life Experience in Type 1 Spinal Muscular Atrophy

Introduction: Nusinersen, an antisense oligonucleotide designed to treat spinal muscular atrophy, led to substantial motor milestone achievements in clinical trials. The aim of this study was to report the clinical outcome of children diagnosed with spinal muscular atrophy type 1 treated with nusin...

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Auteurs principaux: Andreia Lomba, Joana A. Ribeiro, Henriqueta Araújo, Vera Ribeiro, Núria Madureira, Filipe Palavra, Carmen Costa, Isabel Fineza
Format: article
Langue:EN
PT
Publié: Sociedade Portuguesa de Pediatria 2021
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Accès en ligne:https://doaj.org/article/828fb533814c45c68d07e54b9f60eae3
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