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Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

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2012Diagnosing the decline in pharmaceutical R&D efficiency1,978 cited

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2021Approval success rates of drug candidates based on target, action, modality, application, and their combinations113 cited
2021Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases96 cited

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