"A man with an uncommon type of motor neuron disease experienced improved symptoms and continued to work as a physician a year after becoming the first person to receive a drug designed to target the specific genetic mutation that causes his disease. Researchers say the early results are exciting and lay the foundation for more treatments for neurodegenerative diseases caused by rare mutations."
https://www.nature.com/articles/d41586-026-02945-z?utm_source=Live+Audience&utm_campaign=243f0c5b23-nature-briefing-daily-20260917_COPY_01&utm_medium=email&utm_term=0_-33f35e09ea-50276768
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