Wikipedia -WSS

Wikipedia

author：hotspot 2025-08-18 11:30:33 - browse：456

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： Malaria cases in Florida and Texas: Here’s what you need to know
Next article： People can get long Covid without testing positive for virus: Study

explore

WSS

Disclosing genetic information: not as worrisome as once feared

The battle over the Brookhaven Landfill on Long Island

Paxlovid study fails to answer questions over benefit for broader groups

WATCH: Why do fingernails fall off when they're damaged?

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

Paxlovid study fails to answer questions over benefit for broader groups

focus

Wikipedia

This article is exclusive to STAT+ subscribers

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

hotspot

Medicare proposal to cover medical devices draws a mixed reaction

Do masks work? RCTs can't tell us

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

Hospital at home shows how to bring innovation to health care

Guidelines to prevent youth baseball injuries need more muscle

Cloning humans is technically possible. It's curious no one has tried

Heart attack and stroke risk calculators need revising

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

Alkermes shareholders re

3 hurdles to bringing medical devices to the U.S. market

entertainment

Do masks work? RCTs can't tell us

The story of mRNA: From a loose idea to a tool that may help curb Covid

Trans patients at the end of life deserve respect and dignity

In areas with more Black doctors, Black people live longer

WATCH: Why do fingernails fall off when they're damaged?

Hospital at home shows how to bring innovation to health care

entertainment

focus

explore

explore