Informing Dose for Pediatric Rare Diseases—A Survey of Recent Orphan Drugs Approvals
ABSTRACT Collectively, pediatric rare diseases affect millions of children worldwide. Yet, treatment options are limited. Dose selection presents unique challenges in pediatric rare disease drug development. Traditional dose‐finding approaches are impractical for these populations, and conventional...
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| Hauptverfasser: | , , , , , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Wiley
2026-02-01
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| Schriftenreihe: | Clinical and Translational Science |
| Schlagworte: | |
| Online-Zugang: | https://doi.org/10.1111/cts.70503 |
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