hotspot -WSS

hotspot

author：hotspot 2025-09-06 07:17:24 - browse：35611

Duchenne muscular dystrophy Histopathology — Duchenne muscular dystrophy histopathology. Dr. Edwin P. Ewing, Jr./CDC

Japanese drugmaker Nippon Shinyaku said on Monday that its Duchenne muscular dystrophy drug, Viltepso, part of a controversial class of treatments for the rare disease, had failed to reach its primary endpoint in a placebo-controlled confirmatory trial.

Children who received the drug could stand up faster from the floor at the end of the study, but so could children who received a placebo. There was no statistically significant difference between the groups.

Although Nippon did not release detailed data, the failure is notable because Viltepso belongs to a contentious class of drugs called exon-skippers. Duchenne is caused by mutations in a muscle-supporting gene called dystrophin. These drugs are designed to increase the amount of dystrophin protein that patients with specific mutations produce. But the drugs only lead to slight increases in protein levels, and their clinical benefits have never been proven in a large, placebo-controlled study.

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： Jerome Adams on the need for new antivirals for Covid
Next article： Lindsay Lohan welcomes 1st child with husband Bader Shammas

hotspot

Wikipedia

WSS

Dramatically reducing cancer deaths requires the PASTEUR Act

New warning issued for rebooking air travel after delays, cancellations

How dance can help health care workers heal from trauma

Stanford updates investigation of president’s past research

The kidney, in all its complexity, is captured in a new atlas

Stanford updates investigation of president’s past research

focus

hotspot

This article is exclusive to STAT+ subscribers

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

hotspot

What does generative AI mean for health care? We asked experts

Gilgo Beach murders: What questions remain following Rex Heuermann's arrest?

Activists slam private health insurers over coverage restrictions

India sets sights on home

New air purifier tech could be key to improving indoor air quality

Norman Reedus' Daryl Dixon

Alkermes shareholders re

Nearly two

Heart attack and stroke risk calculators need revising

Lindsay Lohan welcomes 1st child with husband Bader Shammas

hotspot

Microsoft strikes partnership with Duke Health to advance health AI

How dance can help health care workers heal from trauma

Couple fighting to bring baby home from Mexico are finally reunited

Plane’s emergency slide plummets from sky, hits Chicago home

Influencer to be charged after chaos erupts in New York City's Union Square

DOJ, FTC to increase scrutiny of all types of health care mergers

Wikipedia

knowledge

entertainment

Wikipedia