focus -WSS

focus

author：entertainment 2025-09-30 18:17:23 - browse：1162

Peter Marks. -- health coverage from STAT — Peter Marks, Director of the Center for Biologics Evaluation and Research at the Food and Drug Administration. Susan Walsh-Pool/Getty Images

Peter Marks wants drug developers to ask more stupid questions.

It’s part of the top Food and Drug Administration official’s plan to reinvigorate gene therapy, a field that has struggled despite significant technological advances. Some companies are shelving programs or going out of business, even when they have promising data.

The problems are numerous: The diseases are often exceptionally rare, limiting the potential market. Manufacturing at commercial quality is complex and expensive. Proving a drug works can be difficult, because there may be too few patients to run a traditional randomized study.

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

Subscribe Log In

Previous article： Apple is now the first public company to be valued at $3 trillion
Next article： NIH documents show early flaws of $1.6 billion long Covid program

WSS

Achieving telehealth equity for people with hearing loss

Bubonic plague hit France in 1720. Officials dithered. Sound familiar?

Achieving telehealth equity for people with hearing loss

Readout LOUD podcast: ASCO preview, a Duchenne trial failure

Ozempic cut risk of death in diabetes patients with chronic kidney disease

Pharma’s losing bet in New Jersey

Wikipedia

focus

knowledge

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

At ASCO, AstraZeneca reports strong data for breast cancer drug

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

HIV drugs protect against transmission, even in unprotected sex

Continuity nursing helped my family in our darkest PICU moments

ASCO Daily Recap: Safety data from Novartis, survival from Pfizer

Guidelines to prevent youth baseball injuries need more muscle

Change Healthcare can notify patients about the breach, HHS says

How one medical school became remarkably diverse

With trial win, GSK could get blood cancer drug back on market

Wikipedia

At ASCO, pharma leaders speak of advances in 'smart bomb' drugs

Asahi Kasei to buy Calliditas, picking up kidney disease drug

U.S. death rates show how politics are affecting public health

Ozempic cut risk of death in diabetes patients with chronic kidney disease

Achieving telehealth equity for people with hearing loss

The Pandemic Treaty must support building strong health systems

hotspot

knowledge

hotspot

hotspot