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AMO Pharma's lead program, tideglusib (AMO-02), has advanced to Phase 3 for congenital myotonic dystrophy (DM1) after successful Phase 2 results. Other pipeline drugs, such as AMO-04 and diazepinomicin (AMO-01), are in Phase 2 for rare neurodevelopmental diseases including Rett syndrome and Fragile X syndrome. The company focuses on rare pediatric neurogenetic disorders.
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