Long-term uninterrupted enzyme replacement therapy prevents …
Administration of OT-58, an enzyme therapy for HCU, during the first 5 weeks of life rescued KO mice survival by preventing liver disease. Here, we studied the impact of a long-term …
Travere Therapeutics: Enzyme Replacement Therapy - HCU …
2024年8月22日 · Classical HCU is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). The study is designed to determine the safety and …
Classical homocystinuria: From cystathionine beta-synthase …
2020年6月1日 · Engineering and chemical modification of human CBS yielded OT-58, a first-in-class enzyme therapy candidate for HCU. Pre-clinical testing of OT-58 showed its substantial …
Enzyme Replacement Therapy Ameliorates Multiple Symptoms of …
Classical homocystinuria (HCU) is the most common inherited disorder of sulfur amino acid metabolism caused by deficiency in cystathionine beta-synthase (CBS) activity and …
First HCU patients treated in Phase 1/2 of OT-58 | HCU
Orphan Technologies, a company dedicated to helping patients control their homocysteine levels, has announced that the first patients with classical homocystinuria have been treated in a …
9000万美元！新型酶替代疗法治疗罕见代谢疾病被看好，该疗法已 …
2020年10月27日 · OT-58 是 Orphan 的新型酶替代疗法，用于患有同型半胱氨酸尿症（HCU）的患者。 HCU 是蛋氨酸代谢过程中由于胱硫醚β合酶（CBS）缺乏引起的罕见的常染色体隐性 …
罕见病信息网 - raredisease.cn
2020年4月10日 · CBS-HCY-CT-01研究是一项双盲、随机、安慰剂对照的1/2期研究，旨在评估OT-58治疗胱硫醚β合成酶缺陷型同型半胱氨酸尿症12岁患者的安全性、耐受性、药代动力学、 …
罕见病领域新动作，Retrophin收购Orphan Technologies获酶替代 …
2021年3月11日 · OT-58是Orphan的创新酶替代疗法，目前正处于治疗典型高胱胺酸尿症（HCU）的I/II期临床开发阶段，这是一种罕见的代谢紊乱，特征为血浆同型半胱氨酸水平升 …
OT-58 as an Enzyme Replacement Therapy for Patients With …
Phase 1 clinical trials of OT-58, an enzyme replacement therapy (ERT) in development for individuals with CBS deficiency (HCU), has been initiated by Orphan Technologies with sites …
Enzyme replacement prevents neonatal death, liver damage, and ...
2017年8月16日 · Classical homocystinuria (HCU: OMIM 236200) represents the most common inherited defect of sulfur amino acid metabolism . It is a rare disease with a variable global …