The underappreciated role of rare disease in drug development
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Happy Friday! I’ve been thinking about the role of rare diseases in drug development this week. Drug companies are racing to sequence healthy volunteers in millions. But very few care about rare disease patients. As much as a healthy human carrying a beneficial mutation helps with drug development, a rare disease patient carrying a pathogenic mutation equally provide useful information. Rare disease informing drug development is one of my favorite topics. Two striking examples that I have highlighted before on Twitter (and on
The underappreciated role of rare disease in drug development
The underappreciated role of rare disease in…
The underappreciated role of rare disease in drug development
Happy Friday! I’ve been thinking about the role of rare diseases in drug development this week. Drug companies are racing to sequence healthy volunteers in millions. But very few care about rare disease patients. As much as a healthy human carrying a beneficial mutation helps with drug development, a rare disease patient carrying a pathogenic mutation equally provide useful information. Rare disease informing drug development is one of my favorite topics. Two striking examples that I have highlighted before on Twitter (and on