Promising gene therapy delivers treatment directly to brain
When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age. A rare genetic disorder kept her from even lifting her head. Her parents …
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Is mesothelin a key to tackling solid tumors? Some biotech companies are betting that it is
On Target is a recurring feature from STAT that dives deep into the most promising drug targets in oncology. This column is adapted from a new STAT report, “Targeting cancer: …
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Opinion: Biopharma leaders helped save democracy. Now what?
The rule of law must be upheld for free societies and free markets to flourish, and only a well-functioning democracy offers the levers for rational majorities to prevail. Americans support …
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In the search for therapies for solid tumors, companies are turning to a novel target: claudin-6
On Target is a recurring feature from STAT that dives deep into the most promising drug targets in oncology. This column is adapted from the new STAT Report: Targeting cancer: …
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FDA approves Alzheimer’s drug shown to moderately slow cognitive decline in early stages of the disease
The Food and Drug Administration on Friday approved a new Alzheimer’s disease treatment that moderately slows cognitive decline in people with early-stage disease. The drug, called Leqembi, was developed by …
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FDA, Biogen, and an Alzheimer’s drug approval: 8 key takeaways from congressional investigation
Congressional investigators sorted through more than 500,000 pages of documents from the Food and Drug Administration and Biogen to chronicle how a controversial Alzheimer’s disease drug was approved, and how …
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Gene therapy trial shows promise for treating ‘bubble boy’ syndrome. Now comes the hard part
H.T. Begay is a happy kid. He’s smiley, silly, and definitely trying to make you laugh. The four-year-old’s two neat braids of dark hair wave behind him as he runs …
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Pharma wins changes in bill regarding FDA’s accelerated approval
A bill that makes it easier for the Food and Drug Administration to withdraw drugs that were approved under an accelerated timeline also dropped provisions the industry had opposed, according …
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We finally have new drugs for sickle cell disease. Why are so few patients taking them?
Related: Companies and researchers are finally pouring resources into a search for better sickle-cell treatments. At first, Lena Harvey didn’t realize anything had changed. She just started doing strange, unexpected …
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