The organizations listed below provide support to patients and families living with Pompe disease through educational information, updates on research and treatment advances, support groups, and more. Please note that the websites listed are maintained by the sponsoring organizations. Sanofi Genzyme is not responsible for maintaining these websites.
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.
A non-profit health organization committed to transforming health through genetics and promoting an environment of openness centered on the health of individuals, families, and communities.
International Pompe Association
The International Pompe Association (IPA) is an International federation of Pompe disease patients groups.
Muscular Dystrophy Association (MDA)
A non-profit health organization dedicated to providing comprehensive medical services to individuals affected by neuromuscular diseases. Pompe disease is one of the more than 40 neuromuscular diseases covered by MDA.
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.
To learn more about the Pompe Registry
LUMIZYME® (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).
WARNING: Risk of anaphylaxis, hypersensitivity and immune-mediated reactions, and risk of cardiorespiratory failure
Anaphylaxis and Hypersensitivity Reactions: Life-threatening anaphylaxis and hypersensitivity reactions have been observed in some patients during and after treatment with alglucosidase alfa. If anaphylaxis or severe hypersensitivity reactions occur, immediately discontinue infusion and institute appropriate medical treatment. Appropriate medical support and monitoring measures should be available during infusion.
Immune-Mediated Reactions: Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs.
Risk of Acute Cardiorespiratory Failure: Patients with acute underlying respiratory illness and compromised cardiac and/or respiratory function may be at risk of acute cardiorespiratory failure. Caution should be exercised when administering alglucosidase alfa to patients susceptible to fluid volume overload. Appropriate medical support and monitoring measures should be available during infusion and some patients may require longer observation times.
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 alglucosidase alfa infusion.
Risk of Antibody Development: As with all therapeutic proteins, there is potential for immunogenicity. There is some evidence to suggest that some patients who develop high and sustained IgG antibody titers may experience reduced clinical efficacy. Patients should be monitored for IgG antibody formation every 3 months for 2 years and then annually thereafter.
The most frequently reported adverse reactions (≥ 5%) in clinical trials were hypersensitivity reactions and included: anaphylaxis, rash, pyrexia, flushing/feeling hot, urticaria, headache, hyperhidrosis, nausea, cough, decreased oxygen saturation, tachycardia, tachypnea, chest discomfort, dizziness, muscle twitching, agitation, cyanosis, erythema, hypertension/increased blood pressure, pallor, rigors, tremor, vomiting, fatigue, and myalgia.
Pregnancy: Based on animal data, alglucosidase alfa may cause fetal harm.
Please see the Full Prescribing Information for complete details, including boxed WARNING.