By collecting clinical information from people living with Pompe disease, the Pompe Registry may ultimately contribute to earlier diagnosis and intervention, and better understanding of disease management.
The Pompe Registry is a global, observational database sponsored by Sanofi Genzyme. The Registry protects the confidentiality of patient information by assigning a unique identification number to each patient such that no patient names are collected.
The Registry provides a repository that allows for the exchange of information and aggregate data to facilitate clinical decision making and data reports, and serves as a vehicle for collaborative studies.
The primary objectives of the Pompe Registry are:
To learn more, visit the Pompe Registry website.
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.