Homocystinuria
Pipeline by Development Stage
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (5)
Total enrollment: 222 patients across 5 trials
A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
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
Homocystinuria: Treatment With N-Acetylcysteine
Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine
Phase Legend
Key Insights
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