focus -WSS

focus

author：comprehensive 2025-09-29 16:21:22 - browse：4

Ionis Pharmaceuticals, a California biotech that has spearheaded the development of RNA-targeting medicines, announced on Tuesday that its drug against familial chylomicronemia syndrome, a rare and serious disease that prevents the body from breaking down fats, succeeded in a late-stage clinical trial.

The Phase 3 trial, dubbed Balance, enrolled 66 adults who were randomly divided into three groups and given a monthly injection of placebo or one of two doses of olezarsen, the company’s experimental drug. Treatment with the higher dose of the therapy significantly lowered triglycerides in the blood of those on the drug for six months compared to participants given placebo, though a lower dose did not have this effect. Participants on the high dose didn’t have any instances of painful inflammation of the pancreas, one of the disease’s most serious symptoms, while there were 11 such episodes among those on placebo.

Researchers reported that the treatment was also generally safe, with fewer adverse events among those on olezarsen than among the placebo group.

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

Subscribe Log In

Previous article： FDA approval of Sarepta Duchenne genetic therapy gives me hope
Next article： NIH chief: government has fallen behind pharma on clinical trials

WSS

Timeline: Key moments in fight for gay rights

Biogen nominates mother of director’s child to replace him on board

After decades in use, heart stents finally beat placebo

Mom launches Girl Scout troop for LGBTQ youth in Texas

Experimental asthma app from Apple and Anthem shows promise

Readout Newsletter: Zepbound, Valneva's vaccine, and more

knowledge

focus

entertainment

George Santos makes 1st court appearance after pleading not guilty to 13 counts

How to fix the rural physician shortage in the U.S.

Dobbs anniversary: the lost opportunity of abortion as health care

A letter from the executive editor: STAT UnitedHealth investigation

Over 800 arrested across France in 3rd night of riots after fatal police shooting of teen

Canadian wildfire dangers should prompt more proactive mitigation from government: Experts

New air purifier tech could be key to improving indoor air quality

What health policies could hitch a ride on a spending bill?

Novavax promises a turnaround & Lilly roils the obesity market

UnitedHealth sued over use of algorithm in Medicare Advantage plans

focus

Ventyx halts psoriasis drug development after lackluster trial results

NIH chief: government has fallen behind pharma on clinical trials

U.K. agency declines to back Eli Lilly’s Mounjaro for type 2 diabetes

NIH chief: government has fallen behind pharma on clinical trials

Eli Lilly’s chief scientist on obesity drugs, gene editing, and more

Cancer diagnosis changes perspective doctor, former hospital CEO

leisure time

entertainment

entertainment

虽然建立新的合作关系和搭建新模式的过程可能相当缓慢，但是我们相信，数据丰富的大环境将增强支付方改变的决心。