We are a biopharmaceutical company focused on developing our pipeline of product candidates including fosmetpantotenate for pantothenate kinase-associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood, and sparsentan for focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN), disorders characterized by progressive scarring of the kidney often leading to end-stage renal disease. Research in additional rare diseases is also underway, including a joint development arrangement evaluating the potential of CNSA-001 in phenylketonuria (PKU), a rare genetic metabolic condition that can lead to neurological and behavioral impairment.learn more
Retrophin is a biopharmaceutical company focused on discovering, developing and marketing innovative therapies for debilitating and often life-threatening diseases. We are dedicated to working in areas in which the biopharmaceutical industry has, to date, had limited interest or effectiveness. We maintain a focus on patients, working with doctors and patient advocacy groups and seeking to understand their distinct needs.