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Adamas Pharmaceuticals, Inc. engages in discovery and development of chrono-synchronous therapies for chronic neurologic disorders. Its products include GOCOVRI, ADS-5012, ADS-4101, and Namzaric. GOCOVRI is indicated for the treatment of dyskinesia in patients with Parkinson's disease receiving levodopa-based therapy. ADS-5102 is a treatment for walking impairment in patients with multiple sclerosis. ADS-401 treats partial onset seizures in patients with epilepsy. Namzaric is a drug treatment for moderate to severe Alzheimer's disease. The company was founded by Gregory T. Went on November 15, 2000 and is headquartered in Emeryville, CA.

Eton Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on developing, acquiring, and commercializing pharmaceutical products for rare diseases. The company offers ALKINDI SPRINKLE, a replacement therapy for adrenocortical insufficiency in children under 17 years of age; Carglumic Acid for the treatment of acute and chronic hyperammonemia due to N-acetylglutamate Synthase deficiency; Betaine Anhydrous for the treatment of homocystinuria; and Nitisinone for the treatment of tyrosinemia type 1. It also provides Zeneo hydrocortisone autoinjector for the treatment of adrenal crisis. Eton Pharmaceuticals, Inc. was incorporated in 2017 and is based in Deer Park, Illinois.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Strongbridge Biopharma Plc operates as a commercial-stage biopharmaceutical company, which focuses on the development and commercialization of therapies for rare diseases. Its pipeline is comprised of Keveyis, Macrilen, Recorlev, and Veldoreotide. The company was founded in 1996 and is headquartered in Trevose, PA.