Hacker News Reader

The disease itself is incredibly expensive. The price of breakthrough treatments will come down faster than the costs of having the disease.

View on HN

I don’t know the specifics of the costs of caring for one with DMD, but I strongly agree that in general, new disease therapies are the only sure way to drive down medical costs in the medium and long term. This represents a victory, no doubt.

It is very expensive. We will likely have $2M USD in insurance claims for medical this year, and our son is currently aged out of the approved label. At a gross cost of $3.2M for this treatment, its expensive but definitely cost effective relatively speaking.

Considering this new treatment is just a single dose[^1] that hopefully lasts for quite a while[^2], it might actually cost a lot less in the long run.

[^1] see FDA press release and monograph. addditional doses won't do much because by then the immune system has learned to attack the AAV vector -- a different vector strain would be needed and that would be a new drug to be approved

[^2] no idea what the turnover rate is. and honestly, the FDA isn't even sure it helps with anything, hence conditional.

Reply on news.ycombinator.com