“Development of a Patient Identification Algorithm to Estimate Prevalence of Classical Homocystinuria in the United States (US)

Insights from the First Genetic Evaluation of a Longitudinal Natural History Study in Classical Homocystinuria (HCU)

Population-Based Incidence Estimates of Classical Homocystinuria Using the Genome Aggregation Database (gnomAD)

Insights from the First Genetic Evaluation of a Longitudinal Natural History Study in Classical Homocystinuria (HCU)

Population-based incidence estimates of classical homocystinuria using the Genome Aggregation Database (gnomAD)

A Longitudinal Study of Cognitive Function in Classical Homocystinuria Demonstrates Distinct Deficits in Inhibitory Control

Pegtibatinase, an Investigational Enzyme Replacement Therapy for the Treatment of Classical Homocystinuria: Initial Results from the Phase 1/2 Compose Study

Dietary Goals and Current Challenges in the Management of Classical Homocystinuria: Insights From Multinational Real-World Experience