Miglustat Shows Promise in Stabilizing Vision in Juvenile Batten Disease
• Treatment with Batten-1 (miglustat) for 18 months showed no signs of deteriorating vision in patients with juvenile Batten disease.
• The Phase 1/2 study demonstrated that Batten-1 was safe and well-tolerated over two years, with potential stabilization of visual acuity.
• A Phase 3 trial, with visual acuity as the primary endpoint, is planned to support drug registration in the U.S. and Europe.
• Batten-1 acts by blocking glucosylceramide synthase, aiming to reduce fatty aggregate buildup in cells.
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