Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook

Anneliene Hechtelt Jonker,Virginie Hivert,Michela Gabaldo,Liliana Batista,Daniel O’Connor,Annemieke Aartsma-Rus,Simon Day, Ken Sakushima,Diego Ardigo

NATURE REVIEWS DRUG DISCOVERY（2020）

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摘要

The International Rare Diseases Research Consortium (IRDiRC) has created a Guidebook to facilitate drug development for rare diseases by organizing available tools into a standardized framework.

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