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Human Induced Pluripotent Stem Cells in Rare Neurological Disorders: Opportunities and Challenges for Disease Modeling and Drug Discovery. [PDF]
Harej Hrkać A +8 more
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Antibody–Drug Conjugates: Smart Chemotherapy Delivery Across Tumor Histologies
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Disentangling the Cost of Orphan Drugs Marketed in the United States
Healthcare, 2023Enrique Seoane-Vazquez +2 more
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Orphan Drug Development in Cardiovascular Medicine
Circulation: Cardiovascular Quality and Outcomes, 2020Javed Butler +2 more
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Flexibility in the FDA Approach to Orphan Drug Development
Nature Reviews Drug Discovery, 2017Rachel E Sherman
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Clinical Pharmacology as a Cornerstone of Orphan Drug Development
Nature Reviews Drug Discovery, 2011Edward Bashaw, Angela Y Men
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Worldwide Collaboration for Orphan Drug Designation
Nature Reviews Drug Discovery, 2016Bruno Sepodes +2 more
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