Drug repurposing for rare: progress and opportunities for the rare disease community

• Published in: Frontiers in medicine Vol. 11; p. 1352803
• Main Authors: Jonker, Anneliene Hechtelt, O'Connor, Daniel, Cavaller-Bellaubi, Maria, Fetro, Christine, Gogou, Maria, 't Hoen, Peter A C, de Kort, Martin, Stone, Heather, Valentine, Nivedita, Pasmooij, Anna Maria Gerdina
• Format: Journal Article
• Language: English
• Published: Switzerland Frontiers Media S.A 2024
• Subjects: drug repurposing; innovation; Medicine; orphan drugs; rare diseases; therapy development; unmet need; innovation; rare diseases; drug repurposing; orphan drugs; unmet need; therapy development

Repurposing is one of the key opportunities to address the unmet rare diseases therapeutic need. Based on cases of drug repurposing in small population conditions, and previous work in drug repurposing, we analyzed the most important lessons learned, such as the sharing of clinical observations, rea...