News for Healthier Living
Breakthrough Gene Therapy Shows Hope for Treating Fatal Childhood Leukodystrophy
Myrtelle Inc. ("Myrtelle"), a pioneering clinical-stage gene therapy company developing transformative treatments for neurodegenerative diseases, today announced the publication of interim results in Nature Medicine from a pioneering clinical study , reporting by researchers the first-in-human application of a gene therapy designed to restore myelin in the brains of children affected by Canavan Disease (CD), a rare and fatal pediatric leukodystrophy. The investigational treatment, known as MYR-101, uses a novel viral vector (rAAV-Olig001) with unique tropism for oligodendrocytes, the brain's myelin- producing cells.
September 16, 2025
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Potential Research Efficiency Gains
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