leisure time -WSS

leisure time

author：explore 2025-09-04 02:30:23 - browse：4888

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： Psychedelics group wrestles with new pharma identity
Next article： Ransomware attacks on hospitals: Study outlines patient impact

entertainment

WSS

World's first CRISPR

Childhood cancer progress stalls for Black, Hispanic kids: Report

Why medical students are struggling with mental health

Morphosys mixed study results for blood cancer drug

Hospitals owned by private equity see complications spike 25%

Editas, Vertex strike deal on CRISPR

hotspot

leisure time

leisure time

Medicare details structure of new drug price negotiation program

Childhood cancer progress stalls for Black, Hispanic kids: Report

Hepatitis C has a cure — but many Americans still lack access to it

Drinking less cuts risk of oral cavity, esophageal cancer: Report

Wildfire smoke exposes gaps in outdoor worker protections

Humana’s Medicare Advantage lawsuit should be tossed, DOJ says

Supreme Court strikes down use of affirmative action

In Spain, a hospital brews its own CAR

How to save PrEP access — and even expand it

Readout LOUD podcast: A CRISPR milestone, biotech mixology

focus

Readout Newsletter: The latest on Moderna, Apellis, and Merck

Medicare drug price negotiation hasn't dried up venture capital

In key trial, Verve's treatment lowers cholesterol levels in patients

Readout Newsletter: What is Casgevy? Does Wegovy hurt muscle?

What STAT readers think about the FDA and patient advocacy

PBM reforms clear House panel

knowledge

entertainment

focus

knowledge