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Mirum Pharmaceuticals Inc is a biopharmaceutical company focused on the identification, acquisition, development, and commercialization of novel therapies for debilitating rare and orphan diseases. Its product, Livmarli, a novel, orally administered, minimally-absorbed ileal bile acid transporter IBAT) inhibitor (IBATi), is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). It is involved in the development of Maralixibat for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, or PFIC, and Alagille syndrome, or ALGS. The company is also involved in the development of volixibat for the treatment of adult patients with cholestatic liver diseases.
Amicus Therapeutics Inc is a biotechnology company focused on discovering, developing and delivering medicines for people living with rare metabolic diseases. The company is engaged in developing a pipeline of medicines for rare metabolic diseases, including a rare disease gene therapy portfolio. It has a portfolio of product opportunities, including oral precision medicine for people living with Fabry disease who have amenable genetic variants; a clinical-stage, treatment paradigm for Pompe disease, and a rare disease gene therapy portfolio. The company has one segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases.