Additional Information

Below are various professional medical organizations, national and international Gaucher disease groups, Gaucher disease education websites, and patient assistance programs that work to support Gaucher disease patients and their families and caregivers.

 

 All information and resources provided by these organizations are operated and maintained by the entity listed below. Please note this list is meant to serve as a general reference and additional resources may also be available through other organizations. With the exception of the specific Sanofi Genzyme* resources listed below, Sanofi Genzyme does not endorse any particular organization or the content and programs they offer.

 

Caregiver Action Network | caregiveraction.org

Caregiver Action Network (CAN) is a family caregiver organization working to improve the quality of life for Americans who care for loved ones with chronic conditions, disabilities, disease, or the frailties of old age. CAN (the National Family Caregivers Association) is a non-profit organization providing education, peer support, and resources to family caregivers across the country free of charge. 

 


Courageous Parents Network (CPN) | courageousparentsnetwork.org

Courageous Parents Network (CPN) is a non-profit organization created to support, guide and strengthen families as they care for sick children. CPN (Courageous Parents Network) web site provides space for parents to learn from other parents and pediatric care providers across the country as they navigate caregiving and advocate for their loved ones.

 


Gauchercare.com* | gauchercare.com

A Sanofi Genzyme-sponsored web site created for people living with Gaucher, their friends and families, and health care professionals. Sanofi Genzyme is committed to helping address the needs of people living with Gaucher disease and those who care for them.

 


Genetic Alliance | geneticalliance.org

Genetic Alliance engages individuals, families, and communities to transform health through genetics and provides opportunities to be active participants in health, from services in traditional health settings to groundbreaking research endeavors. They partner with individuals and communities to transform health systems, convene powerful networks, deliver actionable information, build intuitive tools, and drive policy decisions. Genetic Alliance is a 501 (c)(3) not-for-profit organization.


Genetic Disease Foundation | geneticdiseasefoundation.org

The Genetic Disease Foundation (GDF) was established in 1997 by patients and families affected by genetic disorders. The Foundation’s mission is to support research, education and the prevention of genetic diseases. The foundation is completely run by volunteers, which enables it to use all of its fundraising dollars to directly fight genetic diseases. GDF is a non-profit 501c(3) organization.

 


National Gaucher Foundation (NGF) |gaucherdisease.org

The National Gaucher Foundation (NGF) is an independent nonprofit dedicated to serving U.S. patients with Gaucher disease and their families. Through financial support, educational programming, patient services, and collaboration with medical professionals, NGF empowers Gaucher patients to live a better today. The NGF is a 501 (c)(3) not-for-profit organization.

 


National Organization for Rare Disorders (NORD) | rarediseases.org

NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them.  NORD, along with its more than 260 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services. NORD is a non-profit 501(c)(3) organization.


Patient Access Network Foundation (PAN) |panfoundation.org

The Patient Access Network (PAN) Foundation is dedicated to helping federally and commercially insured people living with life-threatening, chronic and rare diseases with the out-of-pocket costs for their prescribed medications. Partnering with generous donors, healthcare providers and pharmacies, PAN provides the underinsured population access to the healthcare treatments they need to best manage their conditions and focus on improving their quality of life. PAN is an independent, national 501 (c)(3) organization.


Patient Services, Incorporated (PSI) |patientservicesinc.org

Patient Services Inc. (PSI) finds solutions to the challenges that face the chronically ill in the United States. PSI provides financial support and guidance for qualified patients with specific, rare chronic diseases. PSI assistance programs help patients and their families living with an expensive chronic illness afford lifesaving treatment. Patient Services is a 501(c)(3) non-profit, charitable organization.


Sanofi Genzyme Co-Pay Assistance Program* | Call 1 (800) 745-4447, option 3

The Sanofi Genzyme Co-Pay Assistance Program helps eligible individuals in the United States who are prescribed a Sanofi Genzyme treatment with their drug related out-of-pocket expenses, including co-pays, co-insurance, and deductibles, regardless of financial status. This program is sponsored by Sanofi Genzyme. 

 


Society for Inherited Metabolic Disorders | simd.org

The Society for Inherited Metabolic Disorders (SIMD) aims to increase knowledge of and promote research in inborn errors of metabolism in humans and to stimulate interactions between clinicians and investigators in inborn errors of metabolism. The Society seeks to foster training and maintenance of a core of qualified investigators and practitioners in the field and to promote public understanding of inborn errors of metabolism. The SIMD is a non-profit professional organization.

 


*Program/website is owned and operated by Sanofi Genzyme


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 Sanofi Genzyme at 800-745-4447, option 2

Please see Full Prescribing Information (PDF).