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Afraxis is discovering and developing drugs to treat rare and neglected diseases through the modulation of p21-activated kinase (PAK). Afraxis' initial indication of interest is Fragile X syndrome with expansion into other diseases of the central nervous system, including schizophrenia and autism spectrum disorders. PAK is also implicated in cancer, including Neurofibromatosis, a rare form of cancer for which there are no approved drugs. Inhibiting PAK activity has the potential to induce significant anti-tumor activity.