March 22, 2021
Obsessive-Compulsive Symptoms as a Manifestation of Homocystinuria
December 4, 2020
A Sisters Quest For A Cure:
Margie McGlynn, MBA '83 and BS '82, president of the Homocystinuria Network America, has endowed a professorship in the School of Pharmacy and Pharmaceutical Sciences to find holistic solutions for rare genetic diseases like the one that took the lives of two of her sisters in childhood.
November 16, 2020
Retrophin Announces Corporate Name Change to Travere Therapeutics, Inc.
October 22, 2020
Retrophin Announces Agreement to Acquire Orphan Technologies
May 1, 2020
Interview with Dr. Ken Maclean
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
July 30, 2018
Aeglea BioTherapeutics Announces Fall 2018 Medical Conference Schedule
Preclinical Efficacy Data of AEB4104 - a New Enzyme Approach - for the treatment of Homocystinuria
February 28, 2018
Early Detection Key to Best Treatment of Rare Diseases: Expert
August 23, 2017