entertainment -WSS

entertainment

author：explore 2025-08-10 11:42:29 - browse：219

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： DIEP, the 'gold standard' of breast reconstruction, is under threat
Next article： ASCO Takeaways: Winners, snoozers, and the art of cancer care

focus

WSS

Illumina board agrees to spin off Grail, as divestment plans proceed

Can medical device makers keep up in the race to treat obesity?

Illumina board agrees to spin off Grail, as divestment plans proceed

UnitedHealth CEO's Medicaid comments send insurer stocks diving

An outdated Medicare rule harms dementia patients with anxiety

Elevance PBM president out as customers face prescription chaos

hotspot

entertainment

This article is exclusive to STAT+ subscribers

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

entertainment

At least 13 dead in Texas as scorching temps continue

Millions may soon lose Medicaid when they don't have to

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

CMS got Medicare Advantage quality rating wrong, judge rules

Peter Hotez and the public health issue of online harassment

ASCO Daily Recap: CAR

Guidelines to prevent youth baseball injuries need more muscle

Eli Lilly’s Mounjaro succeeds in second weight

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

Can medical device makers keep up in the race to treat obesity?

leisure time

Finland to offer bird flu vaccine to select groups of people, a possible global first

Inside the rise of 'mukbang' eating videos

CMS got Medicare Advantage quality rating wrong, judge rules

Mom launches Girl Scout troop for LGBTQ youth in Texas

Mom launches Girl Scout troop for LGBTQ youth in Texas

Correcting Robert F. Kennedy Jr.'s vaccine 'facts'

leisure time

focus

focus

focus