entertainment -WSS

entertainment

author：Wikipedia 2025-09-27 22:49:58 - browse：92184

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： Alkermes shareholders re
Next article： Disappointing trial forces Pfizer to pivot on obesity medicine plans

WSS

Democratic lawmaker calls for 'well

Flagship Pioneering launches a new biotech in two countries

'The Exorcist' director William Friedkin dead at 87

BIO selects John Crowley as new CEO

UnitedHealth’s Optum accused of antitrust violations in California

Elon Musk says he may need surgery before proposed 'cage match' with Mark Zuckerberg

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+.

hotspot

Hollywood unions extend contract negotiations for actors

Disappointing trial forces Pfizer to pivot on obesity medicine plans

Drug repurposing or repositioning? The language matters

Elon Musk says he may need surgery before proposed 'cage match' with Mark Zuckerberg

23andMe had bad news about my health. I wish a person had delivered it

Israel demolishes home of alleged Palestinian attacker, fueling West Bank tensions

Walensky prepares to leave CDC — and Congress — behind

Biden will tout long

DIEP, the 'gold standard' of breast reconstruction, is under threat

Arizona Democrat to raise extreme heat with Biden 1 year out from Senate primary

explore

Rep. Raul Ruiz on the physician shortage and more

Judges halt a Biden rule offering student debt relief for those alleging colleges misled them

Trump Jan. 6 attorney John Lauro is ex

South Korea begins evacuating thousands of Scouts from coastal campsite as tropical storm nears

DeSantis and Newsom agree to Sean Hannity debate, then disagree on the rules

Takeda, Janssen join wearable maker to measure scratching

focus

entertainment

hotspot

entertainment