fashion -WSS

fashion

author：knowledge 2025-09-27 23:32:40 - browse：7

The FDA building -- First Opinion coverage from STAT — Adobe

U.S. regulators on Wednesday approved the first treatment for an ultra-rare disease that causes people to grow bone where it otherwise shouldn’t be, extending an option to patients who have been advocating for access to the medicine.

The treatment, an oral medication that will be marketed as Sohonos, faced some questions about the quality of its data, and was rejected earlier this year by European regulators. But in approving the drug for the treatment of fibrodysplasia ossificans progressiva, or FOP, the Food and Drug Administration concluded that Sohonos’ benefits outweigh its risks.

“I just closed my eyes and looked up to the skies, like ‘Thank you, God,’” said Suzanne Hollywood, whose teenage son, Joey, has been receiving the drug in a clinical trial. “It’s far from perfect, but I’m thrilled for my son, for his future, and for the kids getting diagnosed who might not have to experience what others had to.”

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED Log In

Previous article： Anesthesiologist group: stop taking Ozempic before surgery
Next article： Annovis Bio delivers candor, but not confidence, on Parkinson’s study

knowledge

WSS

Christmas in the PICU brings some surprising joy

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

He's a Nobel laureate. Critics say he was misleading on Covid

Dario to buy mental health app maker Twill in cash and stock deal

In Spain, a hospital brews its own CAR

STAT Health News: Apple Watch import ban, Pfizer fumbles, etc

Wikipedia

fashion

entertainment

FDA approval of Sarepta Duchenne genetic therapy gives me hope

Medicare drug price negotiation hasn't dried up venture capital

Trump demands the U.S. pay no more for drugs than other countries … again

Prime Medicine, Myeloid Therapeutics clash over genome

As demand for Covid shots wanes, Moderna seeks its next success

Medicare Advantage beneficiaries sue Humana over algorithm use

Duchenne breakthrough therapy leaves behind pioneering families

Hospitals owned by private equity see complications spike 25%

State Dept. review finds Biden bears some blame for Afghanistan failures

HCA sued by state AG for quality issues at Mission Health

knowledge

The Harvard botanist who searched for psychedelics in the Amazon

How to keep neuroscience’s past racism from being its future

David Ricks of Eli Lilly: best biopharma CEO of 2023

Why palliative psychiatry is worth discussing

Editas, Vertex strike deal on CRISPR

David Ricks of Eli Lilly: best biopharma CEO of 2023

knowledge

hotspot

focus

focus