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Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company that is committed to revolutionalizing the care for patients that are living with rare genetic diseases of obesity. It is a company that specializes in developing molecule therapies targeting metabolic pathways for treating rare genetic diseases of obesity, diabetes and gastrointestinal functional disorders.
It focuses on the melanocortin-4 receptor (MC4R) pathway, which is saddled with the responsibility of regulating weight and hunger. By targeting the MC4R pathway, it aims to develop therapeutics for rare genetic diseases of obesity, including Proopiomelanocortin (POMC) Deficiency Obesity, Leptin Receptor (LEPR) Deficiency Obesity, Bardet-Biedl Syndrome (BBS), Alström Syndrome, POMC or LEPR Heterozygous Deficiency Obesity, Smith-Magenis Syndrome, SRC1 Deficiency Obesity, MC4R Deficiency Obesity, and SH2B1 Deficiency Obesity.
The company is leveraging the Rhythm Engine that comprised of its TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program to improve the understanding, diagnosis and potentially the treatment of these disorders.
