entertainment -WSS

entertainment

author：knowledge 2025-10-01 18:22:52 - browse：71

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： The cancer drug shortage isn’t new — and neither are the solutions
Next article： Chamber of Commerce, DOJ clash in court over drug prices

explore

WSS

Boeing's 1st astronaut flight bumped into next year, more repairs needed

Bankrupt Pear's prescription app helped MassHealth save money, data suggests

Prince Harry and Meghan to work on film adaptation of 'Meet Me at the Lake' novel

Trump lawyer welcomes Pence testifying in Jan. 6 trial, won't say if Trump will too

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

FDA weighs limits of animal data in assessing artificial wombs

explore

entertainment

This article is exclusive to STAT+ subscribers

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

Wikipedia

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

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

After affirmative action ruling, medical educators look to 'holistic review'

Information blocking fines are now fair game in health care

Drug repurposing or repositioning? The language matters

Grad students accuse University of California of wage theft

Tori Bowie and the unacceptable rates of Black maternal death

Illinois to ban advertising for guns allegedly marketed to kids and militants

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

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

knowledge

Mississippi candidates for statewide offices square off in party primaries

House panel takes first step toward fixing drug shortages

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

'The Exorcist' director William Friedkin dead at 87

Information blocking fines are now fair game in health care

House panel takes first step toward fixing drug shortages

explore

focus

hotspot

hotspot