1RAOKADAYDec 21, 2022
hilzoy

Good news: a hitherto lethal disease can now be more or less cured.

Bad news: the disease is so rare that no one wants to commercialize the therapy.

Good news: regulatory structures can be fixed more esily than children's missing immune systems. But will we?

RT @[email protected]

Gene therapy trial shows promise for treating ‘bubble boy’ syndrome. Now comes the hard part https://trib.al/aD3KuL7

🐦🔗: https://twitter.com/statnews/status/1605704178801819651

Gene therapy trial shows promise for treating 'bubble boy' syndrome. Now comes the hard part

As gene therapies for rare diseases find success, more cracks are appearing in the current regulatory and commercialization models.

STAT
Dec 21, 2022 at 11:41pmMastodon Twitter Crossposter
Show threadjoel hanesDec 22, 2022

@hilzoy
There once was a moderately-effective vaccine for Lyme disease.

It didn't make much money.

Now there is no vaccine for Lyme disease.