Wikipedia -WSS

Wikipedia

author：knowledge 2025-10-06 17:54:30 - browse：2224

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： State Dept. review finds Biden bears some blame for Afghanistan failures
Next article： Buoyed by wins on obesity, Novo Nordisk eyes heart disease

WSS

Childhood cancer progress stalls for Black, Hispanic kids: Report

Congress must reauthorize a committee to support older Americans

Readout Newsletter: Bayer's future, Merck Caraway acquisition, etc

U.K. considers world's first use of meningitis vaccine to address gonorrhea

Medicare Advantage enrollment grows 7.1% to 33.4 million people

Weight loss drugs aren’t covered by Obamacare. That may change

entertainment

Wikipedia

entertainment

Duchenne breakthrough therapy leaves behind pioneering families

Hospitals look to new technology to keep health care workers safe

How to save PrEP access — and even expand it

Poor hospice wound care hurts patients and families

Hepatitis C has a cure — but many Americans still lack access to it

AbbVie CEO Gonzalez to step down after a decade fiercely defending Humira

Virginia high school admissions case could be legal follow

Readout Newsletter: Sarepta, Alnylam, and Royalty Pharma

Moonlake's readout produced a cash windfall. Risks remain

Poor hospice wound care hurts patients and families

focus

Medicare Advantage enrollment grows 7.1% to 33.4 million people

Hospitals look to new technology to keep health care workers safe

Abridge raises $150m as clinical notes automation market heats up

Abridge raises $150m as clinical notes automation market heats up

Wegovy, Zepbound and weight loss drugs that preserve muscle

Astellas still sees hope in its gene therapy after four deaths

hotspot

focus

hotspot

Wikipedia