Why hemophilia gene therapies have so far been met with crickets

New treatments for hemophilia are here at long last. But where are the patients?

March 13, 2024 Check out STAT+

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Adobe STAT+ | Hemophilia gene therapies arrived after 40 years of struggle. Where are the patients? Researchers spent 40 years and billions of dollars developing gene therapies. But now that they're approved, many patients don't want them. By Jason Mast STAT+ | Hospitals struggle to validate AI-generated clinical summaries. 'It's a bit chaotic' AI-powered summarization tools can save time and angst for hospital staff, but they can also introduce mistakes, or make things up. By Katie Palmer and Casey Ross STAT+ | AI health care companies say they'll keep humans in the loop. But what does that actually mean? The "human in the loop" is meant to be a backstop to prevent flawed AI models from causing harm. But does it work? By Mohana Ravindranath courtesy Gwen Petersen After Amylyx ALS trial failure, patients share disappointment and fear — but some hope ALS patients worry failed trial could impact future drug development, fear loss of insurance coverage. And yet they're clinging onto hope. By Annalisa Merelli Opinion: Good health care price transparency rules are at risk if Congress doesn't act Recently adopted health care price transparency rules are at risk of getting bogged down by regulatory malaise. By Benjamin Chartock STAT+ | Pharma loses a key battle over a state law concerning 340B pharmacies The pharmaceutical industry argued that hospitals that rely on contract pharmacies are abusing a U.S. drug discount program. By Ed Silverman

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