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Tuesday, Oct 31 2023

Full Issue

FDA Panel Meeting Today To Review Proposal For Sickle Cell Gene Therapy

A FDA advisory panel will hear proposals Tuesday from Vertex Pharmaceuticals and CRISPR Therapeutics in an all-day meeting. USA Today examines the potential and high cost of using gene therapy to treat diseases.

Several companies are pursuing gene therapies to treat sickle cell. A federal advisory panel will review the first of those proposals in an all-day meeting on Tuesday.聽Dr. Jeffrey Glassberg, who has had several patients go through gene therapy trials, said the results were impressive. "These people simply don't have sickle cell anymore," said Glassberg, a hematologist at the Icahn Sinai School of Medicine聽at Mount Sinai in New York. (Weintraub, 10/31)

M&Ms, Reese鈥檚, gummy bears, and even cotton candy made an appearance at the Food and Drug Administration on the day before Halloween. Commissioner Robert Califf was not throwing a costume party. Regulators were debating the potential benefits and pitfalls of 鈥渃andy-like鈥 nonprescription drug products such as gummies, particularly for children who often cannot, or do not want to, swallow actual pills. (Florko, 10/30)

Cassava Sciences has long claimed its experimental drug, called simufilam, slows the cognitive decline of people with Alzheimer鈥檚. On Friday, we learned how: The company recruited a large number of people into its clinical trial who don鈥檛 have Alzheimer鈥檚. People who almost certainly had Alzheimer鈥檚 were also included in the study, but in this group, a placebo outperformed Cassava鈥檚 drug. (Feuerstein, 10/30)

The claim: Republican presidential candidate Vivek Ramaswamy ... urged the U.S. to declare 鈥減harmaceutical independence,鈥 saying that 鈥95% of over-the-counter pharmaceuticals are coming from our enemy in China.鈥 PolitiFact ruling: False. The 95% figure is greatly exaggerated. Available data puts the share at no bigger than 20%, and possibly even lower. (Jacobson and Putterman, 10/30)

麻豆女优 Health News: An Arm And A Leg: John Green Vs. Johnson & Johnson (Part 2)

The final episode of this two-part series about YouTube star John Green and his fight to make tuberculosis drugs more affordable takes listeners halfway around the world to India.聽For nearly two decades, activists there have been organizing for patent reform. Host Dan Weissmann and producer Emily Pisacreta speak with one of them, drug patent expert Tahir Amin, about how legal victories in India (and some extra pressure from John Green鈥檚 online community of fans) have set the stage for generic manufacturing and lower-priced TB drugs.聽聽 (10/31)

This is part of the Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.
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