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October 25, 2019

Data from Orphan Technologies' prospective natural 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 receiving care at centers of excellence still experience ocular deficits, inflammation, and metabolic and liver dysfunction
  • Many diagnosed and treated patients have severe cognitive impairment and increased skeletal fragility despite young age

April 9, 2019

Cure for an orphan disease?

First human clinical trial is latest CU Anschutz-led milestone on homocystinuria

February 6, 2019

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 homocysteine levels in preclinical models
  • Ongoing natural history study to define progression of disease in patients suffering from homocystinuria

January 25, 2019

First recipient of the CBS deficiency global grants program announced

HCU Network America and HCU Network Australia today announced the first recipient of their CBS deficiency global grants program – awarding a research grant to the University of Colorado School of Medicine in Denver to explore a potential treatment for homocystinuria due to cystathionine beta-synthase (CBS) deficiency. The research, led by Professor Kenneth N. Maclean aims to investigate the potential usage of different metabolic strategies designed to increase the efficacy of betaine to treat CBS deficient homocystinuria (HCU). Dr. Maclean, is a professor of pediatrics at the University of Colorado School of Medicine, and has had a longstanding interest and involvement in homocystinuria research.

December 7, 2018

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 fails to capture the vast majority of homocystinuria cases

October 10, 2018

Aeglea BioTherapeutics to Present New Interim Phase 1/2 Clinical Trial Data in Arginase 1 Deficiency at 2018 ASHG Annual Meeting

Positive Preclinical Efficacy Data on its AEB4104 Homocystinuria Therapeutic Program