Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals.

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Title: Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals.
Authors: Fletcher EP; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA., Kim C; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA., Mei H; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA., AbuAsal B; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA., Samuels S; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA., Madabushi R; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA., Ramamoorthy A; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA.
Source: Clinical and translational science [Clin Transl Sci] 2026 Feb; Vol. 19 (2), pp. e70503.
Publication Type: Journal Article
Journal Info: Publisher: WileyBlackwell Pub Country of Publication: United States NLM ID: 101474067 Publication Model: Print Cited Medium: Internet ISSN: 1752-8062 (Electronic) Linking ISSN: 17528054 NLM ISO Abbreviation: Clin Transl Sci Subsets: MEDLINE
Database: MEDLINE Ultimate
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ISSN:1752-8062
DOI:10.1111/cts.70503