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First treatment for Alexander disease.

1 report so far. New developments are added to this thread as they happen.

  1. Sep 3, 2026
    FDA APPROVES FIRST-EVER DRUG FOR THE DISORDERFDA approval · 54 patients

    FDA approves first drug for Alexander disease, a rare brain disorder

    A spinal injection given every three months is now the first approved treatment aimed at the cause of Alexander disease, a genetic brain disorder that affects fewer than one in a million people.