leisure time -WSS

leisure time

author：Wikipedia 2025-09-29 22:52:08 - browse：6875

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： Medical leaders decry Supreme Court decision on affirmative action
Next article： Readout Newsletter: Bluebird, CVS, Contineum

WSS

Ukraine taking heavy casualties 10 weeks into its counteroffensive

Living with Hashimoto's and celiac disease, and med school, too

Readout Newsletter: Bluebird, CVS, Contineum

5 to watch: Tech startups trying to solve health care's staffing crisis

Clinical trials must include more disabled people

Intermittent fasting comes with a heart risk? Not so fast

explore

leisure time

focus

Heart attack and stroke risk calculators need revising

Special counsel raises new conflict of interest concerns for De Oliveira's lawyer

Guidelines to prevent youth baseball injuries need more muscle

Gene editing the human germline: What are the risks?

Guidelines to prevent youth baseball injuries need more muscle

An interview with Robert F. Kennedy Jr. on vaccines

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

Nurses recount patients attacks

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

Ukraine taking heavy casualties 10 weeks into its counteroffensive

focus

Trump's false claims about Georgia's 2020 election long debunked

Clinical trials must include more disabled people

Cancer drug shortages' outsize impact on ovarian cancer patients

CVS Caremark has created a new ploy: the drug ‘rebate credit’

Readout Newsletter: Geron, Novo Nordisk, WuXi updates

ActiGraph execs on goal of drug approvals with digital endpoints

entertainment

focus

hotspot

knowledge