Rob Stein
👤 SpeakerAppearances Over Time
Podcast Appearances
In a paper published in the New England Journal of Medicine, two top FDA officials describe what they call a new, quote, plausible mechanism pathway for approving treatments.
Instead of requiring complicated and expensive studies testing an experimental treatment on patients, the FDA says some therapies could be approved based on other criteria that could include evidence that the treatment can help patients by fixing the underlying cause of the disease, like a genetic defect.
The approach is aimed at making it more practical to use cutting-edge technologies like gene editing to treat patients suffering from rare diseases.
You have to cut back on everything.
I mean, I think it's the way it's going to be for a while.
experimental gene editing drug into 15 patients to test whether a one-time infusion can permanently lower cholesterol by editing a gene in the liver, and found the infusion could safely cut cholesterol, as well as triglycerides, by as much as half.
The findings, presented at the American Association's annual scientific meeting, mirror those produced by a similar experimental approach also being tested.
but much more research is needed to confirm that a one-time infusion can safely and effectively cut cholesterol for life.
Doctors infused an experimental gene editing drug into 15 patients to test whether a one-time infusion can permanently lower cholesterol by editing a gene in the liver, and found the infusion could safely cut cholesterol, as well as triglycerides, by as much as half.
The findings, presented at the American Association's annual scientific meeting, mirror those produced by a similar experimental approach also being tested.
but much more research is needed to confirm that a one-time infusion can safely and effectively cut cholesterol for life.
An experimental gene editing drug into 15 patients to test whether a one-time infusion can permanently lower cholesterol by editing a gene in the liver and found the infusion could safely cut cholesterol as well as triglycerides by as much as half.
The findings, presented at the American Association's annual scientific meeting, mirror those produced by a similar experimental approach also being tested.
But much more research is needed to confirm that a one-time infusion can safely and effectively cut cholesterol for life.