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Orphan Technologies Receives Rare Pediatric Disease Designation from FDA for OT-58 to Treat Homocystinuria caused by Cystathionine B-synthase Deficiency
Rare metabolic disorder with no adequate treatment options Causes skeletal abnormalities, osteoporosis, neurodevelopmental delay, and ophthalmologic abnormalities primarily affecting children LEXINGTON, Mass.–(BUSINESS WIRE)–Orphan Technologies, a ... » Read More
Orphan Technologies Publishes Study Showing Prevalence of Classical Homocystinuria Substantially Higher Than Previously Published Estimates
Lexington, Mass.–(BUSINESS WIRE)–Orphan Technologies, a company dedicated to helping patients control their homocysteine levels, published study showing that 12,113 patients in the United States are ... » Read More
Data From Orphan Technologies’ Prospective Natural History Study in Classical Homocystinuria Demonstrates That Current Treatments do not Prevent Severe Disease Outcomes
Orphan Technologies presented 3 Posters at SSIEM 2019 demonstrating: Current standard of care does not adequately control homocysteine levels in patients with classical homocystinuria Patients ... » Read More
Orphan Technologies Announces First Patients Treated in Phase 1/2 Trial of OT-58 in Homocystinuria
Rare metabolic disorder causes severe cardiovascular, skeletal, neurologic, and ophthalmic consequences with no adequate treatments OT-58 is a novel, engineered enzyme has demonstrated reduction in ... » Read More
Orphan Technologies Presents Data Indicating That the Prevalence of Homocystinuria is Substantially Higher Than Previously Estimated
Data presented at ISPOR Europe 2018 demonstrates that the prevalence of homocystinuria (HCU) is greater than that of phenylketonuria (PKU) Study demonstrates that newborn screening ... » Read More
Orphan Technologies to Develop New Treatment for Life-Threatening Metabolic Disorder from the University of Colorado
Licensing and collaboration agreements enable development of enzyme replacement therapy for Homocystinuria, a rare metabolic disorder. AURORA, Colo., February 20, 2013 – The University of ... » Read More