Article

Implementation status of pharmacological studies in the development of orphan drugs

2023-09-05

Abstract excerpt

<title>Abstract</title> <p><bold>Background:</bold> The nonclinical as well as clinical development of orphan drugs is difficult, owing to unknown pathophysiology and the absence of model animals. Both, the U.S. Food and Drug Administration (FDA) Guidance and European Medicines Agency (EMA) Guidelines, for orphan drug development describe non-clinical studies, but lack specific information, such as animal species...

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Literature Corpus work: 59c6ee4e-760e-59f6-9ca6-1b543eb95f9f
DOI: 10.21203/rs.3.rs-3174402/v1

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