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Drug Name:


Generic Name and Formulations:
Betaine anhydrous 1g; per scoopful; pwd for dilution and oral administration.

Rare Disease Therapeutics, Inc.

Therapeutic Use:

Indications for CYSTADANE:

For the treatment of homocystinuria (eg, cystathionine beta-synthase [CBS] deficiency, 5, 10-methylenetetrahydrofolate reductase [MTHFR] deficiency, cobalamin cofactor metabolism [cbl] defect) to decrease elevated homocysteine blood levels.

Adults and Children:

Dissolve one scoop (1g betaine anhydrous pwd) in 4–6 ounces of water, juice, milk, formula, or mixed with food for immediate ingestion. <3yrs: initially 100mg/kg/day divided in twice daily doses, may increase weekly by 50mg/kg increments. ≥3yrs: 3g twice daily. Increase gradually until plasma total homocysteine is undetectable or present only in small amounts. Up to 20g/day has been used.


Hypermethioninemia. Monitor plasma methionine concentrations in patients with CBS deficiency. Pregnancy (Cat.C). Nursing mothers.

Pharmacological Class:

Methylating agent.

Adverse Reactions:

GI upset; hypermethioninemia, cerebral edema.

How Supplied:

Bottle—180g (w. measuring scoop)

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