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Neuren Pharmaceuticals is an Australian biopharmaceutical company focused on developing new therapies for neurodevelopmental disorders with high unmet need. The company's lead product, trofinetide (marketed as Daybue in North America), is the first and only FDA-approved treatment for Rett syndrome, developed in partnership with Acadia Pharmaceuticals. Neuren is also advancing its second lead candidate, NNZ-2591, through Phase 2 clinical trials for several rare genetic conditions, including Phelan-McDermid, Angelman, Pitt-Hopkins, and Smith-Magenis syndromes. The company's therapeutic approach utilizes synthetic analogs of naturally occurring neurotrophic peptides to restore brain cell function and modulate neuroinflammation.
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