>We were able to deliver these treatments to the children in our ongoing clinical trials thanks only to funding from a generous family whose own child is a participant.
Wish this were not the case.
Wish this were not the case.
If it's looked at through the right lens, yes. I'm not sure if genetic research is actually the optimal means to find such takeaways though.
As to whether that means it's an effective payoff if you put more money in rare diseases (and therefore less into non-rare diseases), I can't say, so instead, I have a portfolio with a small but not tiny amount for the collection of all rare diseases.
Lighter regulation around rare "small market" diseases might save a lot of lives. And money.
Orphan drugs for rare diseases are already subject to less regulation.