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by missedthecue 269 days ago
I'm sorry to hear about the passing of your wife.

How common a disease is doesn't have much to do with treatment efforts. Cystic Fibrosis has practically been cured by Big Pharma and only ~40,000 people in the entire US have it.

1 comments

That is because the Cystic Fibrosis foundation funded some of the research and drug development to make the first treatment possible. They had to essentially operate in a VC model to get the treatments that these CF patients need.

https://www.cff.org/about-us/our-venture-philanthropy-model

Most disease categories have advocacy and funding groups, with varying amounts of success. The point is that rare diseases get new treatments all the time from private for-profit firms. In fact, orphan diseases are extremely profitable, and the FDA offers fast tracked approval decisions, making them an enticing niche to focus on.