Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)

计划入组 110 人 · 首次发布于 2016 年 12 月 20 日

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

计划入组 13 人 · 首次发布于 2021 年 12 月 13 日

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

计划入组 70 人 · 首次发布于 2024 年 2 月 7 日

glycogen synthase kinase 3 beta inhibitors (PsychoGenics)

thymidylate synthase inhibitors (INSERM)

Adenylosuccinate Synthase 1(CRD)

Beta Bionics

Beta Innov

France

K-Group Beta, Inc.

United States

Travere Therapeutics Pauses Enrollment in Phase 3 Pegtibatinase Trial for Homocystinuria Due to Manufacturing Issues

FDA Approves Baxfendy (Baxdrostat), a Novel Aldosterone Synthase Inhibitor for Resistant Hypertension

Beyond the Pill: RNA Interference, Aldosterone Synthase Inhibition and Incretins Reshape Hypertension Drug Development