Organizations & Educational Resources

The organizations and online resources listed below can provide information and support to patients and families living with Pompe disease through disease education; updates on research, clinical trials, and treatment advances; support groups and counseling; and more. Please note that the websites listed below are maintained by the sponsoring organizations, Sanofi Genzyme therefore does not control and is not responsible for the content of these websites.

Disease & Patient Organizations

Acid Maltase Deficiency Association (AMDA)
Formed to assist in funding research and to promote public awareness of acid maltase deficiency, another name for Pompe disease, this U.S. organization is a member of the International Pompe Association.

Association for Glycogen Storage Disease (AGSD)
A parent- and patient-oriented support group based in the U.S. The AGSD was established for parents of and individuals with glycogen storage diseases to communicate, share their successes and concerns, share useful findings, provide support as needed, create an awareness of this condition for the public, and to stimulate research in the various forms of glycogen storage diseases.

Genetic Alliance
A nonprofit health advocacy organization committed to transforming health through genetics and promoting an environment of openness centered on the health of individuals, families, and communities.

Muscular Dystrophy Association (MDA)
A non-profit health organization dedicated to providing comprehensive services such as disease information, summer camps, and caregiver resources to individuals affected by neuromuscular diseases including Pompe Disease.

National Organization for Rare Disorders (NORD)
A unique federation of individuals and organizations working together to build a better world for people affected by rare diseases.

United Pompe Foundation
An organization formed to assist patients and/or their families with medical costs and other expenses that may not be covered by insurance. The Foundation also hopes to raise public awareness of Pompe disease.

Pompe Community

This Sanofi Genzyme-sponsored website provides the Pompe disease community with comprehensive information on the disorder, as well as resources and support to help manage the challenges it may bring.

Visit the Pompe Community website

Other Information

A U.S. government database, this website provides a searchable online resource for information on federally and privately supported clinical trials conducted in the United States and around the world.

Food and Drug Administration (FDA)
The United States agency responsible for reviewing and regulating drugs, biologics, and medical devices. The FDA's site is a clearinghouse of information for the public, including new product approvals and safety alerts.

National Society of Genetic Counselors
National Society of Genetic Counselors provides information on the genetic counseling profession and its guiding principles. The website helps you locate genetic counseling services in your area.


LUMIZYME® (alglucosidase alfa) is an enzyme replacement therapy for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).


  • If you or your child is taking Lumizyme, you should know that severe and potentially life-threatening allergic-type reactions known as anaphylaxis or severe hypersensitivity reactions, and other severe reactions related to the immune system may occur during and after Lumizyme treatment. Reactions may include, for example, kidney dysfunction, extreme difficulty breathing, shallow breathing, abnormal heart rate, low blood pressure, or throat tightness (including face, tongue and lip swelling) and skin lesions.
  • If you or your child experiences these severe reactions, you may require close observation during and after Lumizyme administration. You should discuss with your physician the signs and symptoms of anaphylaxis and hypersensitivity reactions and seek immediate medical care should they occur. Anaphylaxis or severe hypersensitivity reactions are potentially very dangerous. If such a reaction is severe enough, your doctor may decide to immediately discontinue the infusion and provide immediate medical care. Appropriate medical support measures may be administered when you are being infused with Lumizyme.
  • If your child has a current respiratory condition or illness and has compromised breathing or heart function due to Pompe disease, there may be risk of acute worsening of heart and/or lung function with Lumizyme due to the fluid of the infusion, and your doctor may decide that close observation during and after Lumizyme administration may be necessary.


Anaphylaxis and Hypersensitivity Reactions: Life-threatening anaphylaxis and hypersensitivity reactions have been observed in some patients during and after treatment with alglucosidase alfa. If such a reaction is severe enough, your doctor may decide to immediately discontinue the infusion and provide you with immediate medical care. Appropriate medical support and monitoring measures should be available during infusion.

Immune-Mediated Reactions: You or your child may be monitored for the development of systemic immune-mediated reactions while receiving Lumizyme. If these reactions occur, your doctor may discontinue the infusion and initiate appropriate medical treatment.

Risk of Acute Cardiorespiratory Failure: Infant Pompe patients with heart or breathing problems who are experiencing an acute respiratory condition may be at risk for increasing the seriousness of these problems as a result of Lumizyme administration due to the infusion fluid, and your child’s doctor may require additional monitoring for these infants.

Risk of Cardiac Arrhythmia and Sudden Cardiac Death during General Anesthesia for Central Venous Catheter Placement: Caution should be used when administering general anesthesia for the placement of a central venous catheter intended for Lumizyme infusion. Ventricular arrhythmias and slow heart rate resulting in cardiac arrest or death have been observed in infant Pompe patients with cardiac hypertrophy during general anesthesia for central venous catheter placement. Appropriate medical support and monitoring measures should be available during infusion.

Risk of Antibody Development: Since Lumizyme is a protein, it is the possible that patients receiving it develop antibodies. Some patients who develop high IgG antibody levels that last for a while may have reduced response to Lumizyme. Patients should be monitored for IgG antibody formation every 3 months for 2 years and then annually thereafter.


The most frequently reported adverse reactions during Lumizyme studies in patients were allergy reactions and included: anaphylaxis, rash, fever, flushing/feeling hot, hives, headache, excessive sweating, nausea, cough, less oxygen in the blood, fast heart rate, rapid breathing, chest discomfort, dizziness, muscle twitching, agitation, bluish or purple skin, redness of skin, high blood pressure/increased blood pressure, facial paleness, chills, tremor, vomiting, fatigue, and muscle pain.


If you are pregnant, you should use Lumizyme only if your doctor has determined that its use outweighs any risks to your unborn child.

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

Please see the Full Prescribing Information for complete details, including boxed WARNING.

Pompe Registry

To learn more about the Pompe Registry, visit