2022
DOI: 10.3233/jnd-221538
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Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative

Abstract: Several successful clinical trials have been conducted in spinal muscular atrophy (SMA) over recent years which have led to the approval of splicing modifiers and gene transfer therapies. With an increasing number of other agents progressing through pre-clinical and clinical development, increasing worldwide clinical trial readiness is becoming essential. SMA Europe initiated a clinical trial readiness project, which included the development of a pilot face-to-face educational-training initiative for clinical … Show more

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