Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics

Rare diseases, affecting millions globally, pose a significant healthcare burden despite impacting a small population. While approximately 70% of all rare diseases are genetic and often begin in childhood, diagnosis remains slow and only 5% have approved treatments. The UN emphasizes improved access...

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Bibliographic Details
Main Authors: Emmanuelle Cacoub, Nathalie Barreto Lefebvre, Dimitrije Milunov, Manish Sarkar, Soham Saha
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-02-01
Series:Frontiers in Public Health
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fpubh.2025.1520467/full
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