Article

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

2024-07-22

Abstract excerpt

<h4>Summary</h4> Rare diseases, affecting millions globally, pose a significant healthcare burden despite impacting a small population. While approximately 70% of all rare diseases are are genetic and often begin in childhood, diagnosis remains slow and only 5% have approved treatments. The UN emphasizes improved access to primary care for these patients and their families. Whole-genome sequencing (WGS) and next-g...

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Literature Corpus work: 5267a977-982c-53f4-a450-60a1f67be7a3
DOI: 10.1101/2024.07.21.24310776

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