Homocystinuria Due to Cystathionine Beta-Synthase Deficiency

DBCOND0144713Congenital, Hereditary, and Neonatal Diseases and Abnormalities Hemic and Lymphatic Diseases

Homocystinuria Due to Cystathionine Beta-Synthase Deficiency is the indication for 1 drug in DrugBank. It is covered by 1 curated, structured indication.

Also known as: Homocystinuria Due to CBS Deficiency
Classified under: Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Hemic and Lymphatic Diseases
Musculoskeletal and Connective Tissue Diseases
Nervous System Diseases
Nutritional and Metabolic Diseases
Urogenital Diseases
Indicated drugs: Betaine

What you can answer from here — as of October 23, 2026

1Indicated drugWith each drug's targets and mechanism

Listed above

1Structured indicationPopulation, line of therapy, route and combination as fields, not prose

1Clinical trialPhase, status and sponsor resolved per trial

1Drug reporting it as an adverse effectWith incidence where the label states it

Which approved drugs for Homocystinuria Due to Cystathionine Beta-Synthase Deficiency share a target, and which companies have late-stage candidates against it?

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