Orphan Technologies is dedicated to helping patients control their homocysteine levels. Our lead program OT-58, is an enzyme replacement therapy that has the opportunity to address the underlying CBS deficit for patients living with homocystinuria.
While patients’ symptoms vary, homocystinuria can increase patients’ risk for cardiovascular, skeletal, ocular, and neuropsychiatric issues. Hallmark symptoms of homocystinuria include ocular symptoms such as lens dislocation or myopia, development delays in children, thrombotic events such as strokes in young adults, or skeletal abnormalities like a sunken chest, lengthening of the long bones, or osteoporosis.
While patients’ symptoms vary, homocystinuria can increase patients’ risk for cardiovascular, skeletal, ocular, and neuropsychiatric issues. Hallmark symptoms of homocystinuria include ocular symptoms such as lens dislocation or myopia, development delays in children, thrombotic events such as strokes in young adults, or skeletal abnormalities like a sunken chest, lengthening of the long bones, or osteoporosis.
— Homocystinuria patient, Age 26
Hear from a homocytninuria patient about the challenges of living with the disease.
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to reducing the burden of patients suffering from homocystinuria.