entertainment -WSS

entertainment

author：fashion 2025-09-29 22:53:23 - browse：66

A Exterior of the new Ultragenyx biomanufacturing plant. -- biotech coverage from STAT — Jonathan Wiggs /Globe Staff

Ultragenyx said Thursday afternoon that its gene therapy for a rare liver ailment, glycogen disease type 1A, succeeded in a Phase 3 trial, setting up a potential approval.

Patients with GSD1a, as the condition is often known, have a genetic mutation that prevents them from adequately maintaining blood sugar levels. Once considered fatal, GSD1a can now be controlled with regular doses of cornstarch. But if patients miss a dose, there can be significant, even life-threatening, complications.

In the 49-person, randomized study, patients who received the Ultragenyx drug were able to take 41% less cornstarch after 48 weeks than they did at the start. Patients on placebo took only 10% less. The difference was statistically significant. Five patients were not included in the analysis, as three dropped out and the company didn’t have 48-week data from two.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Monthly

$39

Totals $468 per year

$39/month Get Started

Totals $468 per year

Starter

$30

for 3 months, then $39/month

$30 for 3 months Get Started

Then $39/month

Annual

$399

Save 15%

$399/year Get Started

Save 15%

11+ Users

Custom

Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users

$300

Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

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

Subscribe Log In

Previous article： United colonoscopy coverage change 'may cost lives,' doctors say
Next article： Obesity now affects over 1 billion people worldwide, study finds

WSS

Wegovy improves function in certain heart failure patients

A Q&A with Aletha Maybank, AMA’s chief health equity officer

Drugmakers bet big on mRNA for cancer. Why's Biden going in?

CDC says it’s too soon to assess risk posed by Covid subvariant

Study: Spinal cord stimulation helps patients with paralysis walk again

International team creates first chimeric human

entertainment

entertainment

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

hotspot

The cancer drug shortage isn’t new — and neither are the solutions

FDA advisory panel to debate high blood pressure devices

Trump demands the U.S. pay no more for drugs than other countries … again

Health officials lay out plans to cope with respiratory virus season

How one medical school became remarkably diverse

California store owner shot dead in dispute over displaying Pride flag: Police

In Memoriam: Notable people who died in 2023

How the drug

Walensky prepares to leave CDC — and Congress — behind

How the first Native Hawaiian psychiatrist helped diversify medicine

hotspot

Former Pear Therapeutics executives start new VC firm

Clinical trials giant ICON acquires HumanFirst

House looks to weaken Medicare negotiation

Trump to be booked at notorious Fulton County Jail at center of DOJ probe

DeSantis wants to import drugs. Biden’s FDA blasted his plan

Covid isolation guidance eased by the CDC

focus

leisure time

focus

hotspot