knowledge -WSS

knowledge

author：knowledge 2025-09-27 15:21:00 - browse：2

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： Peter Hotez and the public health issue of online harassment
Next article： Readout Newsletter: Allogene's pivot, Illumina exec to lead Delfi

explore

WSS

6 surprising facts about sugar

Videos point to link between seizures and unexplained toddler deaths

Ibogaine psychedelic therapy for opioid addiction unlikely, official says

New weight loss drugs work. But how can they best be used?

Ibogaine psychedelic therapy for opioid addiction unlikely, official says

GSK Flovent asthma inhalers replaced by authorized generics

Wikipedia

knowledge

hotspot

Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

Cancer, immunology, HIV research ensnared in fetal tissue politics

As demand for Covid shots wanes, Moderna seeks its next success

U.K. sequencing study surfaces new findings about tumor DNA

What does generative AI mean for health care? We asked experts

Medicare Advantage insurers pressure Biden for bigger payments

Supreme Court strikes down use of affirmative action

Adam's Take: Intra

Heart attack and stroke risk calculators need revising

What Kate Middleton's cancer revelation might teach the world

Wikipedia

Yancopoulos of Regeneron works to expand Dupixent success

GSK Flovent asthma inhalers replaced by authorized generics

Novo Nordisk to buy Cardior, building up pipeline of heart drugs

Federal weight loss drug coverage is 30 years out of date

Bring on the sabbaticals for nurses

Feng Zhang, Grail founder join forces on new gene editing startup

explore

knowledge

focus

leisure time