BH4 (KUVAN) FOR TREATMENT OF SYSTEMIC SCLEROSIS VASCULOPATHY

Systemic Sclerosis (SSc) is a multisystem autoimmune disorder with a progressively devastating course. Increased fibroblast activity results in abnormal growth of connective tissue that causes vascular damage and fibrosis. Fibrosis occurs in the skin, the gastrointestinal (GI) tract, and other internal organs. There is no cure, effective therapy, or gold standard measurement for disease progression. Delay in diagnosis increases morbidity and mortality.

BH4 is a novel approach for treating SSc by targeting vasculopathy that precedes fibrosis. BH4 (KUVAN) is currently approved to treat phenylketonuria (PKU). Short term study results indicate that BH4 (KUVAN) alleviates SSc symptoms in patients.