fashion -WSS

fashion

author：fashion 2025-10-01 21:09:47 - browse：6763

Two people run on the sugar-phosphate backbone of DNA while holding base pairs — in the lab coverage from STAT — Adobe

LONDON — Widespread genetic testing as well as the development of cutting-edge, customized genetic therapies have opened the door to treating many more inherited conditions than previously possible. A few recent cases, in which bespoke medicines were created for children with exceedingly rare mutations, have raised hopes for furthering the approach.

Now, researchers and government agencies in the U.K. are trying to figure out the nitty-gritty to replicate those early one-off successes for as many patients as possible — what sort of preclinical testing a tailor-made treatment needs to go through before being used, what regulatory thresholds each treatment needs to clear, and how to pay for these therapies.

“A lot of the barriers are no longer scientific — we can make these drugs,” said Matthew Wood, a professor of pediatrics at the University of Oxford. “It’s more about, can we make them at a cost that is reasonable enough? And can we get the regulators to reduce the barriers to doing this so that when a patient shows up, you can actually make a drug quickly enough to benefit that patient, rather than it costing huge amounts of money and taking two or three years to do it?”

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Novavax promises a turnaround & Lilly roils the obesity market
Next article： Plane’s emergency slide plummets from sky, hits Chicago home

WSS

Lindsay Lohan welcomes 1st child with husband Bader Shammas

Russia strikes Ukraine's critical port facilities in Odesa after halting grain deal

Readout Newsletter: Novavax stock, FogPharma, Novo Nordisk

Readout Newsletter: Novavax stock, FogPharma, Novo Nordisk

Trump got a 'target letter.' What is one and what does it mean?

Readout Newsletter: Novavax stock, FogPharma, Novo Nordisk

Wikipedia

fashion

knowledge

United colonoscopy coverage change 'may cost lives,' doctors say

More tobacco lobbyists active in statehouses

Psychedelics group wrestles with new pharma identity

Exact Sciences stock tumbles despite strong cancer detection sales

Hollywood unions extend contract negotiations for actors

Health care needs a new profession: neuroimaging counseling

Jerome Adams on the need for new antivirals for Covid

The kidney, in all its complexity, is captured in a new atlas

Virginia high school admissions case could be legal follow

Trump got a 'target letter.' What is one and what does it mean?

entertainment

H5N1 bird flu virus started spreading in cows in Texas in December

Ebola public health emergency is over, WHO declares

Suspected Gilgo Beach killer appeared 'traumatized' after arrest: Defense attorney

Readout Newsletter: Novavax stock, FogPharma, Novo Nordisk

Ebola public health emergency is over, WHO declares

Diversity in clinical trials starts with building trust, experts say

knowledge

focus

hotspot

knowledge