Double duty: Early research reveals how a single drug delivers twice the impact in fragile X

A new study shows how two major pathways — AKT and NMD (nonsense-mediated mRNA decay) — interact in the context of fragile X syndrome. Researchers also found that Afuresertib, a drug currently being tested in phase 1 and 2 clinical trials for several types of cancer, inhibits both pathways in neural stem cells that mimic the disease, leading the cells to act more like typical, non-disease cells.


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Source: ScienceDaily