Rhythm Pharmaceuticals and Axovia Therapeutics sign BBS research collaboration

Rhythm Pharmaceuticals (RYTM) and Axovia Therapeutics announced a joint research collaboration designed to advance the understanding of Bardet-Biedl syndrome, or BBS. BBS is a rare autosomal recessive ciliopathy that presents with a variety of signs and symptoms that evolve over time including visual impairment, renal disease, polydactyly, genital abnormalities, cognitive impairment, hyperphagia and early-onset, severe obesity arising from impairment of the hypothalamic MC4R pathway. In the United States, BBS affects approximately 4,000 to 5,000 individuals with similar prevalence in Europe.