The Pompe Registry, a program sponsored and administered by Sanofi Genzyme, was created in 2001 to gather information to improve the understanding and evaluate the treatment of Pompe disease. It is the largest patient registry dedicated to Pompe disease.
Because Pompe disease is rare, it can be difficult to collect information on how the disease and its treatment affect different people. The Pompe Registry is a global, observational database for gathering this critical information. All people who have Pompe disease are eligible to participate, regardless of whether they are on treatment.
Your physician can enroll you in the Pompe Registry – you cannot enroll yourself. He or she will explain the program and will ask you to sign an authorization to participate in the Registry. Your participation is completely voluntary; however, it may involve long-term follow-up.
When you volunteer to participate in the Pompe Registry, your de-identified medical information is combined with other participants' and is used in studying trends and addressing specific questions about Pompe disease and its treatment.
Patient privacy is a top concern for the Pompe Registry. Information about you can only be submitted following your written authorization. Your name will not be collected by the Registry. Instead, you and all enrolled patients are randomly assigned an identification number. Only your physician will know what number is assigned to you.
Talk with your doctor about enrolling you in the Registry, or visit www.registrynxt.com for more information.
To learn more about the Pompe Registry
LUMIZYME® (alglucosidase alfa) is an enzyme replacement therapy for patients with Pompe disease (acid α-glucosidase (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 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: Patients with infantile-onset Pompe disease (IOPD) should be managed by a clinical specialist knowledgeable in immune tolerance induction in Pompe disease to optimize treatment. Some patients who develop high and sustained IgG antibody levels, including certain IOPD patients, may have a reduced response to Lumizyme.
Monitoring: Laboratory Tests: 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.
Please see the Full Prescribing Information for complete details, including boxed WARNING.