Anneliene Hechtelt Jonker on Muck Rack

Anneliene Hechtelt Jonker

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Articles

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

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By Anneliene Hechtelt Jonker, Virginie Hivert, Michela Gabaldo, Liliana Batista
| Nature Verified
COMMENT 20 April 2020 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. Daniel O’Connor, Annemieke Aartsma-Rus, Simon Day, Ken Sakushima & Millions of people worldwide are affected by rare diseases, but it is estimated that fewer than 5% of the known rare diseases have at least one approved pharmacotherapy1.
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