Article: Transformational therapy cures haemophilia B

https://www.bbc.co.uk/news/health-62240061

 

one less ltc 'needing' cbt.

 

Bold claims, but this reminds me of a prominent BPS ideologue who sarcastically made the observation that for the chronically ill, medical breakthroughs are massively important, while psychosocial progress is derided as nothing.

Yeah, if it can completely cure millions it's definitely massively important. This is real life-changing stuff, if true. Meanwhile a BPS approach to this would have been the usual: suck it up therapy leaves them less "stressed" and that's good enough. Literally worse than nothing because this approach blocks the kinds of efforts like this that can change everything.

 

I think it almost certain that this is slam dunk bona fide.
This is where medicine is really transforming what can be achieved.
If gene editing achieves normal levels of a protein it means not only that the treatment has got to its target but it has harnessed the regulatory mechanisms needed to restore 'normality'.
Cures for one gene diseases like haemophilia have been pretty predictable in the last five years - just a matter of doing the technical work. And the issues about cost in the article look to me trivial. The alternative of constant replacement therapy must be much more expensive in the long run. So this will be a money saver as well.

So, yes, the BPS people can go on playing King Canute but real medicine does move forward when it can.

 

Sounds great. The patients are expected to need treatment every 10 years or so (nobody knows for sure). So quite not a cure, more like a really good, low maintenance treatment.