Phase 1 clinical trials of OT-58, an enzyme replacement therapy (ERT) in development for individuals with CBS deficiency (HCU), has been initiated by Orphan Technologies with sites in the United States. For more information click here.
OT-58 as an Enzyme Replacement Therapy for Patients With Cystathionine Beta-Synthase Deficient Homocystinuria (CBSDH)
