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UniQure eyes UK approval for embattled Huntington’s gene therapy after FDA quarrel

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UniQure plans to submit AMT-130 to the U.K.’s Medicines and Healthcare products Regulatory Agency in the third quarter of 2026 based on Phase 1/2 data showing a 75% slowing of disease—the same data the FDA has deemed unacceptable for a biologics license application.

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STAT+: Where ‘democracy met science,’ 50 years ago

Get your daily dose of health and medicine every weekday with STAT’s free newsletter Morning Rounds. Sign up here.

Good morning. At a Cambridge bar on Saturday, I watched straight-seeming couples congregate by a television showing basketball, while a more queer-coded crowd lingered at another showing soccer. I don’t think that’s anything, really, but it was fun. 

This ‘never event’ is happening more frequently

A child born with congenital syphilis could suffer dire consequences: bone deformities, brain damage, blindness, deafness, and more. But that should be a ‘never event’ as public health officials say: A pregnant person can receive an injectable form of penicillin to prevent the infection. Somehow, rates keep going up anyway. Between 2012 and 2024, the U.S. saw an 800% increase in babies born with the disease. And since last year, there’s been a shortage of the drug.

Continue to STAT+ to read the full story…

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Get your daily dose of health and medicine every weekday with STAT’s free newsletter Morning Rounds. Sign up here.

Good morning. At a Cambridge bar on Saturday, I watched straight-seeming couples congregate by a television showing basketball, while a more queer-coded crowd lingered at another showing soccer. I don’t think that’s anything, really, but it was fun. 

This ‘never event’ is happening more frequently

A child born with congenital syphilis could suffer dire consequences: bone deformities, brain damage, blindness, deafness, and more. But that should be a ‘never event’ as public health officials say: A pregnant person can receive an injectable form of penicillin to prevent the infection. Somehow, rates keep going up anyway. Between 2012 and 2024, the U.S. saw an 800% increase in babies born with the disease. And since last year, there’s been a shortage of the drug.

Continue to STAT+ to read the full story…

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Sanofi wins type 1 diabetes nod for Tzield after requesting to revoke CNPV

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Sanofi makes no mention of the Commissioner’s National Priority Voucher. Tzield was awarded the ticket in October 2025, but Sanofi requested withdrawal from the program after former CDER head Tracy Beth Høeg reportedly expressed skepticism of the drug.

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Rhythm’s obesity drug scores ‘better than expected’ weight loss in rare genetic disease

Rhythm’s obesity drug scores ‘better than expected’ weight loss in rare genetic disease

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Rhythm Pharmaceuticals’ Imcivree reduced fat—while boosting muscle—in patients with Prader-Willi syndrome.​ ​Read More

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