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Evolving landscape of drug development for pediatric rare diseases-from successes to strategies for addressing unmet needs.

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Title: Evolving landscape of drug development for pediatric rare diseases-from successes to strategies for addressing unmet needs.
Authors: Stern S; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Zhang Y; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Pressly M; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Yang X; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Abdel-Rahman S; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Wang J; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Chen MC; School of Pharmacy, College of Pharmacy, Taipei Medical University, 250 Wuxing St., Taipei 110301, Taiwan., Chen CH; Department of Pharmacology, School of Medicine, College of Medicine, Taipei Medical University, 250 Wuxing St., Taipei 110301, Taiwan., Pepe S; Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Doi M; Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Burckart GJ; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Yao LP; Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Schuck R; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA., Bai JP; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA. Electronic address: Jane.Bai@fda.hhs.gov.
Source: Journal of pharmaceutical sciences [J Pharm Sci] 2026 Jun 20; Vol. 115 (9), pp. 104401. Date of Electronic Publication: 2026 Jun 20.
Publication Type: Journal Article; Review
Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 2985195R Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1520-6017 (Electronic) Linking ISSN: 00223549 NLM ISO Abbreviation: J Pharm Sci Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Evolving landscape of drug development for pediatric rare diseases-from successes to strategies for addressing unmet needs.
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  Data: <searchLink fieldCode="AU" term="%22Stern+S%22">Stern S</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Zhang+Y%22">Zhang Y</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Pressly+M%22">Pressly M</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Yang+X%22">Yang X</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Abdel-Rahman+S%22">Abdel-Rahman S</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Wang+J%22">Wang J</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Chen+MC%22">Chen MC</searchLink>; School of Pharmacy, College of Pharmacy, Taipei Medical University, 250 Wuxing St., Taipei 110301, Taiwan.<br /><searchLink fieldCode="AU" term="%22Chen+CH%22">Chen CH</searchLink>; Department of Pharmacology, School of Medicine, College of Medicine, Taipei Medical University, 250 Wuxing St., Taipei 110301, Taiwan.<br /><searchLink fieldCode="AU" term="%22Pepe+S%22">Pepe S</searchLink>; Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Doi+M%22">Doi M</searchLink>; Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Burckart+GJ%22">Burckart GJ</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Yao+LP%22">Yao LP</searchLink>; Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Schuck+R%22">Schuck R</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA.<br /><searchLink fieldCode="AU" term="%22Bai+JP%22">Bai JP</searchLink>; Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, Maryland 20903, USA. Electronic address: Jane.Bai@fda.hhs.gov.
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  Data: <searchLink fieldCode="JN" term="%222985195R%22">Journal of pharmaceutical sciences</searchLink> [J Pharm Sci] 2026 Jun 20; Vol. 115 (9), pp. 104401. <i>Date of Electronic Publication: </i>2026 Jun 20.
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              Text: 2026 Jun 20
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