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Methyl donor (homocystinuria)

Betaine

Brand names: Cystadane

Betaine (betaine anhydrous) is a methyl-group donor used in the treatment of homocystinuria to lower elevated plasma homocysteine concentrations.

Dosing — being independently re-sourced

ClinCalc Pro is rebuilding its dose data from primary open sources — the manufacturer SmPC (eMC), the WHO Model Formulary and other official references — under clinician review. This drug's structured dose is not yet published here. Confirm all doses against the product SmPC and your local formulary before prescribing.

Clinical monograph

How it works

It acts as a substrate for betaine-homocysteine methyltransferase, providing a methyl group that remethylates homocysteine to methionine and so reduces homocysteine levels.

Prescribing in practice

  • Cases of cerebral oedema have been reported, particularly with raised plasma methionine, so monitor and adjust treatment under specialist metabolic supervision.
  • It is used as part of comprehensive management of homocystinuria alongside other measures rather than as monotherapy in isolation.
  • Plasma methionine concentrations can rise during treatment and require monitoring.

Monitoring

Monitor plasma homocysteine and methionine concentrations and watch for symptoms of cerebral oedema under specialist guidance.

Counselling the patient

  • This medicine lowers a harmful amino acid called homocysteine.
  • Attend all blood-test appointments arranged by the metabolic team.
  • Report any severe headache, vomiting or visual changes promptly.

Evidence & guidelines

Betaine is an established treatment for lowering homocysteine in homocystinuria as reflected in specialist metabolic guidance and the SPC.

Reference: BIMDG; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. Monograph status: clinician-reviewed (2026-07-04).

Related

Curated clinical cross-links plus same-class fallbacks.

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