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Clinical Trials for Homocystinuria

• 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
• Safety, Tolerability and Pharmacodynamics of SYNB1353 in Healthy Adult Volunteers
• A Multiple Ascending Dose Study of ACN00177 in Subjects With CBS Deficiency
• Effect of Acetaminophen and N-Acetylcysteine on Liver Metabolism on Homocystinuria
• Pegtibatinase as an Enzyme Therapy for Patients With Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
• Betaine METABOLISM OF PATIENTS With Homocystinuria
• EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment
• Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine
• Homocystinuria: Treatment With N-Acetylcysteine