entertainment -WSS

entertainment

author：Wikipedia 2025-09-06 03:53:38 - browse：15

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： Cancer drug shortages should be causing more outrage
Next article： How neuroscience research could help beat back brain cancer

hotspot

WSS

STAT wants to know your pick for a great summer read or listen

Humans aren't the only ones with an imagination. Rats have it too

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

The wait for US passports is creating travel purgatory and snarling summer plans

Readout LOUD podcast: ESMO highlights & Roivant's big deal

Readout LOUD podcast: ESMO highlights & Roivant's big deal

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

In age of alternative facts, a scholarly course on calling out crap

Beware reading your test results before your doctor does

Moonlake's readout produced a cash windfall. Risks remain

How to convince healthy people to buy insurance

Psychedelics group wrestles with new pharma identity

Canadians with cystic fibrosis outlive Americans by a decade

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

What is microchimerism and how does it affect maternal health?

Apple is now the first public company to be valued at $3 trillion

Explaining an after

entertainment

The health care issue Democrats can’t solve: hospital reform

Brain injuries and the importance of a second opinion

Key Senate panel to consider PBM, drug pricing reform package

Medicare must police algorithms in Medicare Advantage, Dems say

All of Us, NIH's precision medicine initiative, gets a big boost

What is microchimerism and how does it affect maternal health?

entertainment

leisure time

hotspot

entertainment