~~<i id='E7905323FA'><strike id='E7905323FA'><tt id='E7905323FA'><pre id='E7905323FA'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

hotspot

Wikipedia

author：comprehensive 2025-09-29 23:12:26 - browse：5

Sarepta Therapeutics said Monday afternoon that its gene therapy for Duchenne muscular dystrophy failed to improve muscle function compared to a placebo in a large clinical trial — likely a major disappointment for patients and doctors who have been desperately awaiting the treatment for years.

The company said all patients in the study improved and that secondary measurements indicated the drug was having an effect. Nevertheless, the results are likely to raise questions about whether the Food and Drug Administration will expand access to the medicine.

Trading in Sarepta shares was halted at $107 with the release of the study results.

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

leisure time

Wikipedia

entertainment

hotspot

entertainment

Wikipedia

Colorizedscanningelectronmicrographofanapoptoticcell(pink)heavilyinfectedwithSARS-COV-2virusparticle

WSS

Information blocking fines are now fair game in health care

How one medical school became remarkably diverse

Akili changes sales model for its ADHD video game treatment

Duchenne breakthrough therapy leaves behind pioneering families

Moonlake's readout produced a cash windfall. Risks remain

BioMarin wins approval for gene therapy to treat hemophilia A

hotspot

Wikipedia

leisure time

Supreme Court strikes down use of affirmative action

FDA approval of Sarepta Duchenne genetic therapy gives me hope

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

Hollywood unions extend contract negotiations for actors

Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

Why the quest to eradicate polio has stumbled

Peter Hotez and the public health issue of online harassment

Medical records are filled with copy

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

Midwives could help bridge maternal health disparities in the U.S.

Wikipedia

Cancer drug shortages should be causing more outrage

Over 800 arrested across France in 3rd night of riots after fatal police shooting of teen

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

Hollywood unions extend contract negotiations for actors

Tori Bowie and the unacceptable rates of Black maternal death

Wildfire smoke exposes gaps in outdoor worker protections

entertainment

hotspot

entertainment

Wikipedia