hotspot -WSS

hotspot

author：fashion 2025-08-23 18:38:54 - browse：9485

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： Wildfire smoke exposes gaps in outdoor worker protections
Next article： JPM: CVS says Medicare Advantage patients are impacting profits

WSS

Man killed during FBI raid in connection with threats against Biden, other officials

Chronically ill young patients struggle to find fertility info

Amylyx's ALS drug launch starts strong. It could be trouble for FDA

FDA panel issues narrow recommendation for Covid vaccine booster doses

Jack Smith previously got search warrant for Trump's Twitter, new court docs show

Chronically ill young patients struggle to find fertility info

hotspot

hotspot

This article is exclusive to STAT+ subscribers

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

knowledge

Moonlake's readout produced a cash windfall. Risks remain

South Korea throws huge K

New Eli Lilly Alzheimer’s data poses Medicare coverage conundrum

UCB departs biotech trade group BIO

Malaria cases in Florida and Texas: Here’s what you need to know

Methadone doses haven’t kept up in the age of fentanyl

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

3 medical device trends to watch in 2024

Guidelines to prevent youth baseball injuries need more muscle

Videos point to link between seizures and unexplained toddler deaths

hotspot

White House finalizes ITC's import ban of latest Apple Watches

Wegovy’s successful heart trial raises vital question about weight loss

Drastic changes should bring profitability, startup health insurers say

Friends ask where James Watson's attitudes about race came from

Goldman Sachs launches $650M fund for life science investments

Wegovy’s successful heart trial raises vital question about weight loss

hotspot

knowledge

knowledge

focus