entertainment -WSS

entertainment

author：leisure time 2025-10-02 22:58:09 - browse：48463

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： Anesthesiologist group: stop taking Ozempic before surgery
Next article： Gut bacteria may contribute to kidney autoimmunity, study finds

WSS

Scientific mystery highlights the blind spots in genomics databases

Chinese researcher claims birth of first gene

With Wegovy coverage, Medicare patients may face high costs

U.K. officials, researchers try to ease path for bespoke genetic treatments

Medicare Advantage insurers pressure Biden for bigger payments

Adam's Take: Intra

knowledge

entertainment

This article is exclusive to STAT+ subscribers

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

explore

Dobbs anniversary: the lost opportunity of abortion as health care

Texas board proposes rules on abortion law emergency exemption

The high cost of giving birth even with insurance

Young boy dies in trial for Pfizer Duchenne gene therapy

Drug repurposing or repositioning? The language matters

6 surprising facts about sugar

Psychedelics group wrestles with new pharma identity

First Opinion on colorectal cancer screening: letter and response

State Dept. review finds Biden bears some blame for Afghanistan failures

Hundreds died while taking an arthritis drug, but nobody alerted patients

hotspot

Readout Newsletter: Illumina, CRISPR, Regeneron

Pregnancy ages women, but effects reverse after delivery

Depression: FDA OKs Otsuka Pharmaceuticals digital therapeutic

Medicare Advantage insurers pressure Biden for bigger payments

Bird flu reported in person exposed to presumably infected cattle

Readout LOUD podcast, live from the STAT Breakthrough Summit East

Wikipedia

explore

hotspot

explore