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Catalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.

Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.

Mallinckrodt Plc is a global specialty pharmaceuticals company. It develops, manufactures, markets and distributes both branded and generic specialty pharmaceutical products and medical imaging agents. It operates through the Specialty Brands and Specialty Generics segments. The Specialty Brands segment includes branded medicines. The Specialty Generics segment relates to specialty generic drugs, active pharmaceutical ingredients, as well as external manufacturing. The company was founded by Gustavo Mallinckrodt, Otto Mallinckrodt and Edward Mallinckrodt in 1867 and is headquartered in Blanchardstown, Dublin.

ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.