~~<i id='1214DD8E91'><strike id='1214DD8E91'><tt id='1214DD8E91'><pre id='1214DD8E91'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

knowledge

fashion

author：Wikipedia 2025-08-11 09:47:42 - browse：2

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： BioMarin wins approval for gene therapy to treat hemophilia A
Next article： It's time to retire 'poor historian' from clinicians' vocabularies

Wikipedia

hotspot

focus

entertainment

knowledge

Onthisweek’s“ReadoutLOUD,”we’reliveinSanFranciscoatSTAT’sBreakthroughSummitWest.AIisabigthemethisyea

WSS

Addiction experts warn against a second 'war on drugs'

Adidas to release second batch of Yeezy sneakers after breakup with Ye

A pollution tax on older cars can be extended to London's suburbs after a British court ruling

Cytokinetics CEO stresses company can launch heart drug on its own

NVIDIA and Google execs on the future of AI and drug discovery

Case highlights the risks of experimental stem cell therapy

knowledge

fashion

Wikipedia

Anesthesiologist group: stop taking Ozempic before surgery

Microsoft's Peter Lee says ChatGPT shouldn't be used for initial diagnosis

At least 13 dead in Texas as scorching temps continue

Europe is fastest

At least 13 dead in Texas as scorching temps continue

Second House panel advances two

In Memoriam: Notable people who died in 2023

Case highlights the risks of experimental stem cell therapy

Tori Bowie and the unacceptable rates of Black maternal death

Biden has enormous polling lead in Democratic primary, despite challenge from RFK Jr.

hotspot

Venture capitalist Bob Kocher on the state of digital health

American TikTok star stuck in Dubai for months following rental car altercation

Young Chinese opt out of the rat race and pressures at home to pursue global nomad lifestyle

House GOP plan to stem drug shortages differs from Democrats’

Helping hospitals and community health centers plan for resilience

Doctors increasingly discourage vaping amid mounting health concerns

focus

entertainment

knowledge

knowledge