Research - Lexington, Massachusetts, United States
Orphan Technologies is committed to reduce the burden of patients suffering from homocystinuria. OT-58, our lead drug development candidate, has been optimized as an enzyme therapy for classical homocystinuria, a genetic disease characterized by debilitating cardiovascular, skeletal, neurologic, and ophthalmologic complications. OT-58 is designed to reduce homocysteine levels via a targeted mechanism of action and may have therapeutic applications in other diseases.
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