explore -WSS

explore

author：Wikipedia 2025-09-05 03:01:45 - browse：8

Sarepta - Cambridge -AP — Kristoffer Tripplaar/Sipa via AP

To hear Sarepta CEO Doug Ingram tell it, the results Monday afternoon from a large trial of its gene therapy for Duchenne muscular dystrophy marked a clear medical breakthrough. “A massive win,” he told investors, that could lead to its approval for all patients with the fatal muscle-wasting disease regardless of age.

And yet the rest of the Duchenne community was more cautious in its enthusiasm. The study — the most rigorous test to date of a technology 30 years in the making — had technically failed, as so many had before it.

“It’s extremely frustrating,” said John Brandsema, a pediatric neurologist at Children’s Hospital of Philadelphia who worked on the study. “When you spend decades trying to bring therapies into the clinic for something so severe and just keep missing over and over again.”

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： 23andMe had bad news about my health. I wish a person had delivered it
Next article： Bring on the sabbaticals for nurses

focus

WSS

More doctors should go into startup world

Novo Nordisk to buy Cardior, building up pipeline of heart drugs

Free speech concerns arise in wake of journal editor Eisen's firing

Senate panel clears PBM, hopsital reforms

Free speech concerns arise in wake of journal editor Eisen's firing

10 million people disenrolled from Medicaid over past six months

hotspot

explore

focus

FDA approval of Sarepta Duchenne genetic therapy gives me hope

Surgeons perform first xenotransplant using CRISPR'd pig kidney

Bright Health sells Medicare Advantage business to Molina

U.K. officials, researchers try to ease path for bespoke genetic treatments

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

Extreme heat events are a public health emergency

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

'Patient friction coefficient' can gauge a clinical trial's patient burden

Tori Bowie and the unacceptable rates of Black maternal death

U.K. officials, researchers try to ease path for bespoke genetic treatments

Wikipedia

Cancer, immunology, HIV research ensnared in fetal tissue politics

Biotech incubator Curie.Bio graduates its first startup

Bring on the sabbaticals for nurses

Just how powerful is the placebo effect?

CRISPR technology availability, affordability needs to be expanded

Adam's Take: Intra

focus

focus

Wikipedia

explore