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本文引用的文献
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Emergence of pediatric rare diseases: Review of present policies and opportunities for improvement.
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Postmarketing trials and pediatric device approvals.
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Unintended consequences of regulatory initiatives in childhood cancer drug development.
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What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis.
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"Creating hope" and other incentives for drug development for children.
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Priority review vouchers: an inefficient and dangerous way to promote neglected-disease drug development.
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