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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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  Data: <searchLink fieldCode="AU" term="%22Fletcher+EP%22">Fletcher EP</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Kim+C%22">Kim C</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Mei+H%22">Mei H</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22AbuAsal+B%22">AbuAsal B</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Samuels+S%22">Samuels S</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Madabushi+R%22">Madabushi R</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Ramamoorthy+A%22">Ramamoorthy A</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA.
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              Text: 2026 Feb
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