focus -WSS

focus

author：Wikipedia 2025-09-30 17:03:45 - browse：859

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： FDA approval of Sarepta Duchenne genetic therapy gives me hope
Next article： Biotech venture capitalists face new demands from investors

entertainment

Wikipedia

WSS

NIH director takes cautious approach to Biden drug pricing priority

Parkinson’s finding may lead to tests and, someday, treatments

Readout Newsletter: BridgeBio, Madrigal, and Silicon Valley Bank

How digital therapeutics firms are pivoting after Pear's bankruptcy

Gilead’s Trodelvy fails in lung cancer, raises questions on ADCs

Copay coupons to count toward deductibles, after Biden court move

Wikipedia

focus

focus

What does generative AI mean for health care? We asked experts

Humana cuts profit projections as Medicare Advantage costs soar

Wildfire smoke exposes gaps in outdoor worker protections

What to make of Gilead’s Trodelvy troubles and Elevation Oncology’s rise

Drug repurposing or repositioning? The language matters

Prior authorization will have to move faster under new Biden rule

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

Medicare Advantage today, explained in one chart

New air purifier tech could be key to improving indoor air quality

My yearlong odyssey to get my son’s ADHD medication covered

entertainment

eGenesis tests gene

Gilead’s Trodelvy fails in lung cancer, raises questions on ADCs

What to make of Gilead’s Trodelvy troubles and Elevation Oncology’s rise

NIH director takes cautious approach to Biden drug pricing priority

The surgeon had a dilemma only a Nazi medical text could resolve

FDA clears AI tool for detecting skin cancer

Wikipedia

entertainment

Wikipedia

hotspot