AMT-130 Marks a Historic Moment for Huntington’s—Now Comes Careful Communication
On July 10, 2024, I wrote a reflection piece following the investor conference where uniQure presented the encouraging 24-month results of AMT-130, the first HTT-directed gene therapy delivered directly into the brain regions most severely affected by mutant huntingtin —the caudate and putamen. At that time, early evidence suggested the therapy might exert a stabilizing […]
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