Rob Stein
👤 SpeakerAppearances Over Time
Podcast Appearances
It has long been controversial because of questions about how well it works and how safe it is.
The FDA is requiring the drug company Sarepta to include a black box warning, the agency's strictest, on the company's gene therapy for Duchenne muscular dystrophy.
The FDA is also restricting use of the gene therapy to patients who are at least four years old and can still walk.
The FDA's action comes in response to reports of liver failure in some patients who receive the gene therapy.
The treatment is the only gene therapy approved for the devastating muscle disorder.
It has long been controversial because of questions about how well it works and how safe it is.
The NIH put Jenna Norton on indefinite paid administrative leave on her first day back to work from the government shutdown.
Norton, a program director at the National Institute of Diabetes and Digestive and Kidney Diseases, has been a vocal critic of cuts and new policies at the NIH.
The NIH did not give Norton an explanation, but in a statement to NPR, an administration official said, quote,
Radical leftist Jenna Norton chooses to constantly criticize this administration even when she's supposed to be working, unquote.
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.
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.