Gene Therapy Shows Signs of Delayed Progression in Fatal Neurodegenerative Disease

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Source: MedPage Today

Original: https://www.medpagetoday.com/neurology/generalneurology/119830...

Published: Tue, 10 Feb 2026 16:58:15 -0500

Gene therapy shows signs of delayed progression in fatal neurodegenerative disease. A single intravenous infusion of adeno-associated virus serotype 9 encoding beta-galactosidase (AAV9-GLB1) has been associated with short-term adverse effects. The therapy led to increased levels of beta-galactosidase in the body. Signs of delayed disease progression were observed. A study published on MedPage Today confirms these findings in patients with this fatal disease.