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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.
Rhythm Pharmaceuticals Inc is a commercial-stage biopharmaceutical company. It is engaged in the development and commercialization of therapies for patients with rare diseases. The company is focused on its melanocortin-4 receptor (MC4R) agonists, including its lead asset, IMCIVREE (setmelanotide), as a precision medicine designed to treat hyperphagia and severe obesity caused by rare MC4R pathway diseases. Geographically the company generates its revenue from the United States and internationally with the majority being generated from the United States.