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A gene therapy success in Huntington's and Trump's autism announcement

September 26, 2025

Why were doctors and investors so excited about trial results of UniQure's gene therapy for Huntington's disease? Are autism rates actually rising? And what does the research tell us about Tylenol and autism?

We discuss all that on this week's episode of the "The Readout LOUD," STAT's biotech podcast. We bring on UniQure CEO Matt Kapusta to discuss the company's announcement this week that its one-time treatment for Huntington's disease significantly slowed down the neurological condition in a key study.

We also chat about the Trump's administration initiatives on autism. Officials this week warned pregnant women not to take Tylenol, saying that it may cause autism, and they announced an FDA decision to make a decades-old drug called leucovorin available as a treatment for people with certain autism symptoms. To unpack the implications of these statements, we bring on by Ari Ne'eman, an assistant professor of health policy and management at Harvard's school of public health who researches how policies affect people with disabilities.

 

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