New Gene Therapy Slows Huntington’s Disease by 75%

Published on Sky News

A breakthrough gene therapy called AMT-130 has slowed progression of Huntington’s disease by as much as 75% in a Phase 1 trial. Delivered via brain surgery, a single dose introduces functional DNA expected to last a lifetime. Patients remained stable and in some cases regained independence, marking what UCL investigators call a world-changing step toward the first licensed therapy to slow this devastating disorder.

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