This week on "The Readout LOUD" podcast, there was an in-depth discussion about uniQure's AMT-130 gene therapy for Huntington's disease. The therapy is administered directly into the brain as a single injection and uses microRNA to silence the mutated HTT gene, thereby reducing the production of toxic huntingtin. uniQure announced positive topline results from a pivotal phase I/II study in patients with early Huntington's disease. Approximately 75,000 people in the US suffer from this disease. The study included 29 early-stage patients, where a higher dose of AMT-130 slowed disease progression by up to 75% over three years. The therapy is administered stereotactically to the structures of the putamen and the caudate nucleus under magnetic resonance control. The results support the potential to alleviate symptoms and slow disease progression.[1][2][3][4][6]