Records containing: Homocystinuria
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CLINICAL_TRIALS study record update · 2026-02-19A Study To Evaluate The Acceptability Of the Express Plus Range, Foods For Special Medical Purposes (FSMP), In Children And Adults With Inborn Errors of Metabolism (IEM)
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CLINICAL_TRIALS study record update · 2026-02-12Creatine Supplementation for CBS-related Homocystinuria: A Proof-of-concept Study in Healthy Adults
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CLINICAL_TRIALS study record update · 2025-08-12Universal Genomic Newborn Screening in the Wallonia-Brussels Federation: Baby Detect
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CLINICAL_TRIALS study record update · 2025-04-11An Open-Label, Phase 3 Long-Term Extension (LTE) Study To Assess The Safety, Tolerability, And Efficacy Of Treatment With Pegtibatinase In Participants With Classical Homocystinuria (HCU) Due To Cystathionine Beta Synthase Deficiency (ENSEMBLE)
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CLINICAL_TRIALS study record update · 2025-04-10Development and Validation of a Standardized Assessment Instrument for Health-related Quality of Life (HrQoL) in Patients With Classical Homocystinuria
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CLINICAL_TRIALS study record update · 2025-04-09A Phase 3, Parallel-Group Treatment, Blinded, Randomized, Placebo-Controlled Study To Assess The Efficacy And Safety Of Pegtibatinase Administered Subcutaneously In Addition To Standard Of Care In Participants With Classical Homocystinuria Due To Cystathionine Beta Synthase Deficiency (HARMONY)
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CLINICAL_TRIALS study record update · 2025-04-04Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in Newborns
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CLINICAL_TRIALS study record update · 2024-11-22A Double Blind, Randomized, Placebo-controlled, Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effects on Clinical Outcomes of Pegtibatinase (TVT-058), Administered Subcutaneously in Patients with Cystathionine Beta-Synthase Deficient Homocystinuria (COMPOSE)
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CLINICAL_TRIALS study record update · 2023-06-18Introduction of Tandem Mass Spectrometry (MS/MS) Technology in the Program of Selective Screening of Hereditary Metabolic Diseases in Kazakhstan
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CLINICAL_TRIALS study record update · 2023-03-17A Phase 1, Dose-escalation, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, and Pharmacodynamics of SYNB1353 in Healthy Volunteers
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CLINICAL_TRIALS study record update · 2023-02-03Betaine METABOLISM OF PATIENTS With Homocystinuria
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CLINICAL_TRIALS study record update · 2021-03-30Effects of Exercise on Metabolic Parameters in Treated Patients With Classical Homocystinuria: a Research Project on a Small Sample Size, Compared to Healthy Controls.
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CLINICAL_TRIALS study record update · 2018-07-09Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine
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CLINICAL_TRIALS study record update · 2009-02-18Homocystinuria: Treatment With N-Acetylcysteine