4 ms·
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
by missedthecue 1y 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.
- pkaye 1y agoThat 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 https://www.cff.org/about-us/our-venture-philanthropy-model
- missedthecue 1y agoMost 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.