Links

The links listed below will connect you to professional medical organizations, hospitals, national and international Gaucher disease groups, patient support organizations, and medical centers specializing in Gaucher disease care and research.

Gaucher Care
http://www.gauchercare.com
Comprehensive information on Gaucher disease and its management from Genzyme.

National Organization for Rare Disorders, Inc.
http://www.rarediseases.org
A non-profit federation of voluntary health organizations dedicated to helping people with rare "orphan" diseases and assisting the organizations that serve them.

Genetic Alliance
http://www.geneticalliance.org
The world's leading nonprofit health advocacy organization committed to transforming health through genetics.

National Gaucher Foundation (USA)
http://www.gaucherdisease.org
A national non-profit organization that funds research, offers financial assistance, promotes education and awareness, supports legislative issues and provides outreach programs

Gaucher Registry
http://www.gaucherregistry.com
The largest patient registry for Gaucher disease, sponsored and administered by Genzyme.

National Tay-Sachs & Allied Diseases Association
http://www.ntsad.org
An organization established to lead the fight to treat and cure Tay-Sachs, Canavan and related genetic diseases and to support affected families and individuals in leading fuller lives.

Society for Inherited Metabolic Disorders
http://www.simd.org
A non-profit professional organization whose purpose is to promote the worldwide advancement of research and medical treatment of inherited disorders of metabolism.

Society for the Study of Inborn Errors of Metabolism
http://www.ssiem.org
A society created to foster the study of inherited metabolic disorders and related topics, and to promote the exchange of ideas among professionals interested in inherited metabolic disease.

Indication & Usage

Cerezyme® (imiglucerase for injection) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions:

  1. anemia
  2. thrombocytopenia
  3. bone disease
  4. hepatomegaly or splenomegaly

Important Safety Information

Approximately 15% of patients have developed IgG antibodies to Cerezyme during the first year of therapy. Approximately 46% of patients with detectable IgG antibodies experienced symptoms of hypersensitivity, and these patients have a higher risk of hypersensitivity. It is suggested that patients be monitored periodically for IgG antibody formation during the first year of treatment.

Hypersensitivity has also been observed in patients without detectable IgG antibodies. Symptoms suggestive of hypersensitivity have been noted in approximately 6.6% of all patients, and anaphylactoid reactions in less than 1%. Treatment with Cerezyme should be approached with caution in patients who have exhibited hypersensitivity symptoms such as pruritus, flushing, urticarial, angioedema, chest discomfort, dyspnea, coughing, cyanosis, and hypotension. Pre-treatment with antihistamines and/or corticosteroids and a reduced rate of infusion may allow continued treatment in most patients.

In less than 1% of patients, pulmonary hypertension and pneumonia have been observed during treatment with Cerezyme. These are known complications of Gaucher disease regardless of treatment. Patients with respiratory symptoms in the absence of fever should be evaluated for the presence of pulmonary hypertension.

Approximately 13.8% of patients have experienced adverse events related to treatment with Cerezyme. Some of these are injection site reactions such as discomfort, pruritus, burning, swelling or sterile abscess at the site at the site of venipuncture. Additional adverse reactions that have been reported include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and tachycardia. Transient peripheral edema has also been reported for this therapeutic class of drug.

To report suspected adverse reactions, contact Genzyme at 800-745-4447, option 2 or FDA at 800-FDA-1088 or http://www.fda.gov/Safety/MedWatch

Please see Full Prescribing Information (PDF).

Cerezyme is the only therapy with over 17 years of use in over 5,600 Gaucher patients, including children.