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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.
Catalyst Pharmaceuticals Inc is a biopharmaceutical company. It focuses on developing and commercializing, developing therapies for people with rare, debilitating, chronic neuromuscular and neurological diseases. It offers three drug products, FIRDAPSE (amifampridine), FYCOMPA (perampanel), and AGAMREE (vamorolone).