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>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 part
by beams_of_light 5y ago
>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.
- bckr 5y agoI think it's okay to express this, but would be more valuable if you added more to the conversation e.g. what you positively desire.
- inglor_cz 5y agoRare diseases need some public funding. Not only for ethical reasons, but we may also find out very nontrivial things about our own biology this way.
- loeg 5y agoThey have some public funding.
- contravariant 5y agoWe probably shouldn't put the main focus on rare disease but on genetic manipulation in general. It would be much more valuable to be able to cure arbitrary genetic defects than just the ones that are somewhere between uncommon and extant.
- DoreenMichele 5y agoNot only for ethical reasons, but we may also find out very nontrivial things about our own biology this way. 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.
- dekhn 5y agoI mean, this has definitely happened in the past; study of various rare genetic diseases has elucidated and illuminated the processes by which diseases occur at a molecular level, and often have utility far outside the original rare disease. 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.
- mym1990 5y agoIt's weird to say 'only' since I assume that a lot more went into this effort other than funding. There are a lot of initiatives that have a lot of funding, but little success. Either way, this result is certainly better than nothing, or waiting years/decades for public funding.
- Talanes 5y ago'Only' implies that the funding was necessary, not that the funding was fully responsible. Also consider that 'only' was said by the researchers. From their pov the effort is a given, because that is what they control, while the funding is an anomalous outside factor.
- hammock 5y ago
- Shared404 5y agoFrom https://news.ycombinator.com/newsguidelines.html https://news.ycombinator.com/newsguidelines.html : > Please respond to the strongest plausible interpretation of what someone says, not a weaker one that's easier to criticize. Assume good faith.
- deleted 5y ago[deleted]
- hammock 5y agoFair enough. Let's hope some charity decides to take up the cause for those children whose families cannot support the cost of treatment.
- BurningFrog 5y agoI expect most of the cost is regulatory. Lighter regulation around rare "small market" diseases might save a lot of lives. And money.
- soldehierro 5y ago> 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.
- halukakin 5y agoWish governments would spare funding to these research efforts.
- thehappypm 5y agoIt’s just tough because resources are not unlimited. You’ll save many more lives focusing on new antibiotics and then you will on Tay-Sachs. I don’t think there’s enough researchers in the world to focus on every single rare disease.