COMPOSE® Phase 1/2, dose-escalation Cohort 7 design: safety of pegtibatinase in children aged ≥5-<12 years with classical homocystinuria (HCU)
Society for Inherited Metabolic Disorders (SIMD) Annual Meeting 2024
April 14 - 17, 2024
Charlotte
Association Between Homocysteine and Clinical Outcomes in Patients With Classical Homocystinuria: A Systematic Literature Review
Genetic Metabolic Dietitians International (GMDI) Annual Conference 2024
April 17 - 20, 2024
Charlotte
Society for the Study of Inborn Errors of Metabolism (SSIEM) Symposium 2024
September 3 - 6, 2024
Porto
Pegtibatinase: The HARMONY/ENSEMBLE (Phase 3 Studies): Study Design
Classical Homocystinuria: Disease State and Treatment Options
COMPOSE (Phase 1/2 Study): Study Design & Results
Pegtibatinase: Mechanism of Action and Pharmacology
ACAPPELLA Study: Natural History Study of Classical HCU
Validation of a patient identification algorithm to estimate the prevalence of classical homocystinuria (HCU) in the United States (US)
Clinical burden of classical homocystinuria in the United States: a retrospective analysis of Optum Market Clarity
A Quantitative Systems Pharmacology (QSP) Model for Classical Homocystinuria Predicting Efficacy of Treatment