Support Networks

 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 (low red blood cell count)
  2. thrombocytopenia (low blood platelet count)
  3. bone disease
  4. hepatomegaly or splenomegaly (enlarged liver or spleen)

Important Safety Information

Approximately 15% of patients have developed immune responses (antibodies) to Cerezyme during the first year of therapy. These patients have a higher risk of an allergic reaction (hypersensitivity). Your doctor may periodically test for the presence of antibodies. Serious allergic reactions (anaphylaxis) have been reported in less than 1% of patients. Symptoms suggestive of allergic reaction happened in approximately 7% of patients, and include itching, flushing, hives, swelling, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. If you have had an allergic reaction to Cerezyme, you and your doctor should use caution if you continue to receive treatment with Cerezyme.

High blood pressure in the arteries of the lungs (pulmonary hypertension) and pneumonia have been observed in less than 1% of patients during treatment with Cerezyme. These are also known complications of Gaucher disease regardless of treatment. If you experience symptoms such as shortness of breath or chest pain, with or without fever, contact your doctor.

Approximately 14% of patients have experienced side effects related to treatment with Cerezyme. Some of these reactions occur at the site of injection such as discomfort, itching, burning, swelling or uninfected abscess. Other side effects, each of which was reported by less than 2% of patients, include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Temporary swelling in the legs has also been observed with drugs like Cerezyme.

Please see Full Prescribing Information (PDF).

You may report side effects to the FDA at 1-800-FDA-1088.

Reference 1. Data on file. Genzyme Corporation; Cambridge, MA.