Case Study: Dose Escalation in Pediatric Rare Disease Trials
Case Study: Dose Escalation in Pediatric Rare Disease Trials Dose Escalation in Pediatric Rare Disease Trials: A Practical Case Study Background, Objectives, and Population Context This case study describes a first‑in‑pediatrics, multicenter dose‑escalation trial for an oral small molecule intended to up‑regulate a deficient metabolic pathway in a rare autosomal recessive disorder. The syndrome manifests…
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