comprehensive -WSS

comprehensive

author：focus 2025-08-23 08:57:41 - browse：67699

Here is STAT’s biotech scorecard, our regular ledger of stock-moving biotech events, for the fourth quarter:

The Food and Drug Administration’s decision in June to grant conditional approval to a gene therapy for Duchenne muscular dystrophy was a watershed moment for the treatment of the neuromuscular disease, and a big win for its maker, Sarepta Therapeutics. Confirming the gene therapy’s benefit — and potentially broadening its use to include older boys — will be determined by a Phase 3 clinical trial that will read out later this quarter.

Pfizeris developing its own gene therapy for Duchenne in a fully enrolled Phase 3 study with final results expected in 2024. But before the verdict is revealed, Pfizer intends to conduct an interim analysis later this quarter. The most likely outcome of this early look will be a determination to continue the study to its completion. But surprises — including an early stop for overwhelming efficacy — can’t be ruled out.

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

Subscribe Log In

Previous article： Cancer drug shortages should be causing more outrage
Next article： Dogs on Prozac: What pet anti

WSS

H5N1 bird flu found in pasteurized milk: what we know

Celiac disease study examines precisely how gluten triggers injury

Bird flu outbreak in cows is latest avian flu curveball

UnitedHealth financials solid despite Change Healthcare cyberattack

H5N1 bird flu found in pasteurized milk: what we know

Close Medicare's dangerous gaps in addiction treatment coverage

focus

comprehensive

hotspot

Walensky prepares to leave CDC — and Congress — behind

After traumatic brain injury, life support decisions can use more time

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

Cell therapy fails to slow type 1 diabetes, but safety is established

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

Involuntary treatment for addiction doesn't work

Wildfire smoke exposes gaps in outdoor worker protections

Elon Musk says Twitter has 'negative cash flow' and 'heavy debt'

How to save PrEP access — and even expand it

Health care needs a new profession: neuroimaging counseling

knowledge

Who gets to use the title 'doctor'?

Congress likely to miss an opportunity to fix drug shortages issue

Georgia court denies Trump's request to quash DA's 2020 election probe

4 killed in mass shooting in Georgia, shooter remains on the loose: Police

Why police arrested suspected Gilgo Beach killer away from his house

Cancer researcher Catriona Jamieson is sending tumors into space

knowledge

leisure time

hotspot

entertainment