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NNZ-2591 is an investigational synthetic analog of cyclic glycine-proline (cGP), a breakdown product of human insulin-like growth factor 1 (IGF‑1). It has been chemically modified to increase its half-life, stability, and oral bioavailability. NNZ‑2591 is being developed primarily for the treatment of rare neurodevelopmental disorders including Angelman syndrome, Pitt-Hopkins syndrome, Phelan-McDermid syndrome (22q13 deletion syndrome), Prader-Willi syndrome, and Fragile X syndrome. The drug acts as a neuroprotectant and modulator of synaptic transmission and neuropeptide receptors; it is believed to exert its effects by restoring normal communication between brain cells in conditions where this is impaired. Preclinical studies have shown that NNZ‑2591 can normalize behavioral deficits in animal models relevant to these syndromes. The developer has received Orphan Drug Designation from both the FDA and EMA for several indications[2][4][5][7].
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