financetom
Business
financetom
/
Business
/
Pfizer's Duchenne gene therapy fails in late-stage study
News World Market Environment Technology Personal Finance Politics Retail Business Economy Cryptocurrency Forex Stocks Market Commodities
Pfizer's Duchenne gene therapy fails in late-stage study
Jun 12, 2024 3:42 PM

June 12 (Reuters) - Pfizer ( PFE ) said on Wednesday its

experimental gene therapy for Duchenne muscular dystrophy (DMD)

failed to improve the motion function of patients in a

late-stage trial compared with a placebo.

The therapy also did not show a significant difference

compared to placebo in secondary goals of the study such the

time it takes for patients to rise from the floor or improve

speed in a 10-meter run or walk.

DMD is a genetic muscle wasting disorder in which most

patients lack the protein dystrophin, which keeps muscles

intact. The disorder affects an estimated 1-in-3,500 male births

worldwide.

Pfizer's ( PFE ) one-time gene therapy aims to deliver a shortened

version of the missing human dystrophin gene to DMD patient's

cells.

The results of the trial in boys ages 4 to 7 years marks

another setback for Pfizer ( PFE ), which needs revenue from new

medicines as billions of dollars from sales of COVID-19 products

have evaporated due to sharply declining demand.

Pfizer ( PFE ) said last month that a patient in an earlier stage

trial of 2- to 3-year-old boys had died from a cardiac arrest

after receiving the therapy. It said then it was reviewing data

to understand the potential cause of death along with the

independent external data monitoring committee.

The company said it will continue to closely monitor all

participants enrolled in the late-stage study and is evaluating

appropriate next steps for the program.

The main goal of the trial looked for improvements in the

speed and functionality of motor skills in DMD patients who can

walk.

Comments
Welcome to financetom comments! Please keep conversations courteous and on-topic. To fosterproductive and respectful conversations, you may see comments from our Community Managers.
Sign up to post
Sort by
Show More Comments
Related Articles >
GRAPHIC-S&P 500 correction in six charts
GRAPHIC-S&P 500 correction in six charts
Mar 15, 2025
NEW YORK, March 13 (Reuters) - For the first time in over a year, the U.S. stock market is in a correction. The question now is whether the slide is set to get worse. The benchmark stock index closed down more than 10% from its February 19 closing high, meeting the widely used definition of a correction. The S&P 500's...
China's Baidu launches two new AI models as industry competition heats up
China's Baidu launches two new AI models as industry competition heats up
Mar 15, 2025
HONG KONG, March 16 (Reuters) - China's Baidu ( BIDU ) said on Sunday it has launched two new artificial intelligence models, the multimodal ERNIE 4.5 and a new reasoning-focused model called X1, as it vies to stand out in a fiercely competitive AI race. Chinese AI startup DeepSeek's roll-out of AI models which it says is on par with,...
China's Baidu launches two new AI models as industry competition heats up
China's Baidu launches two new AI models as industry competition heats up
Mar 15, 2025
HONG KONG (Reuters) - China's Baidu ( BIDU ) said on Sunday it has launched two new artificial intelligence models, the multimodal ERNIE 4.5 and a new reasoning-focused model called X1, as it vies to stand out in a fiercely competitive AI race. Chinese AI startup DeepSeek's roll-out of AI models which it says is on par with, or even...
Voice of America staff put on leave, Trump ally says agency 'not salvageable'
Voice of America staff put on leave, Trump ally says agency 'not salvageable'
Mar 15, 2025
(Reuters) - -More than 1,300 Voice of America employees were placed on leave on Saturday and funding for two U.S. news services that broadcast to authoritarian regimes was terminated, one day after President Donald Trump ordered the gutting of the government-funded media outlet's parent and six other federal agencies. Michael Abramowitz, Voice of America's director, said nearly his entire staff...
Copyright 2023-2026 - www.financetom.com All Rights Reserved