Resources for Patients

Downloadable information for your patients and families to learn more about the genetic disorders that cause homocystinuria and instructions on how to take CYSTADANE.

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Living with Classical Homocystinuria

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Living with MTHFR deficiency

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Living with a Cobalamin Cofactor Metabolism Defect

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Education and Advocacy

The following educational and advocacy groups provide information about rare genetic disorders, including the different types of homocystinuria. You can explore these groups online.

HCU Network America

The mission of HCU Network America is to help people with homocystinuria (HCU) and related disorders manage their disease and to find a cure.

Organic Acidemia Association

The Organic Acidemia Association is a patient advocacy organization that provides support and information for people with inherited metabolic disorders. Homocystinuria caused by several cobalamin defects—cblC, cblD, cblF, cblJ, and cblX—is included as a part of the group’s advocacy activities.

The National Organization for Rare Disorders (NORD)

NORD is a patient advocacy organization dedicated to helping people with rare diseases. NORD provides information on homocystinuria due to CBS deficiency.

INDICATIONS AND USAGE

Cystadane is a methylating agent indicated in pediatric and adult patients for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations. Included within the category of homocystinuria are:

  • Cystathionine beta-synthase (CBS) deficiency
  • 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency
  • Cobalamin cofactor metabolism (cbl) defect

IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia in Patients with CBS Deficiency: CYSTADANE may worsen elevated plasma methionine concentrations and cerebral edema has been reported. Monitor plasma methionine concentrations in patients with CBS deficiency. Keep plasma methionine concentrations below 1,000 micromol/L through dietary modification and, if necessary, a reduction of CYSTADANE dosage.
  • Most common adverse reactions (> 2%) are: nausea and gastrointestinal distress, based on physician survey.
  • To report SUSPECTED ADVERSE REACTIONS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1‑800-FDA-1088 or www.fda.gov/medwatch.
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IMPORTANT SAFETY INFORMATION

  • Hypermethioninemia in Patients with CBS Deficiency: CYSTADANE may worsen elevated plasma methionine concentrations and cerebral edema has been reported. Monitor plasma methionine concentrations in patients with CBS deficiency. Keep plasma methionine concentrations below 1,000 micromol/L through dietary modification and, if necessary, a reduction of CYSTADANE dosage.
  • Most common adverse reactions (> 2%) are: nausea and gastrointestinal distress, based on physician survey.