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FTY720-Mitoxy is a research-stage small molecule and synthetic derivative of the FDA-approved multiple sclerosis drug fingolimod (FTY720). It was designed as a mitochondrial-targeted stimulator of protein phosphatase 2A (PP2A) activity to treat neurodegenerative diseases such as Parkinson's disease and Multiple System Atrophy (MSA). Unlike fingolimod, FTY720-Mitoxy is not phosphorylated in vivo, which prevents it from modulating sphingosine-1-phosphate receptors (S1PRs) and avoids the systemic immunosuppression associated with the parent drug. Preclinical studies indicate that the compound crosses the blood-brain barrier, increases the expression of neurotrophic factors (BDNF, GDNF, NGF), reduces α-synuclein pathology, and restores mitochondrial function.
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