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. |
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| 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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