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VGD is an inherited metabolic disorder in which a shortage of glucose-6-phosphatase enzyme prevents glycogen being converted into glucose, leading to an accumulation of glycogen in the liver and kidneys. The symptoms of VGD include fasting intolerance, low blood sugar, and poor growth. XNX741 is designed to use a novel molecule inside nanocells to provide controlled conversion of glucose to glycogen in the absence of glucose-6-phosphatase.
Tsukuhara syndrome leads to a curved spine, small head, and premature fusion of the bones in the skull. Mental retardation occurs in many patients. XNX459 is designed as combined device-drug therapy to treat mental deficits that occur in patients with Tsukuhara syndrome.