Supportive Care

Managing Pompe disease usually involves a variety of supportive care to address a patient's specific symptoms. Because the disease can cause a wide range of symptoms, patients are best followed by a team of physicians across different medical areas, led by a care coordinator with experience treating Pompe disease or similar disorders.

Depending on your particular symptoms and experiences, your multidisciplinary care team may include several different specialist healthcare providers, such as neuromuscular specialists, metabolic specialists, pulmonologists, cardiologists, physical therapists, speech therapists, and dieticians. As a patient, it is important to communicate with all the specialists involved in your medical care. You can also play a role in encouraging these specialists to communicate amongst themselves and regularly with the physician leading your care.

Ongoing Monitoring

Pompe disease is a multi-system disorder, meaning that it can affect many of the body's systems and organs. The effects of Pompe disease vary widely from person to person, and because of the disorder's progressive and unpredictable nature, regular patient monitoring is important to:

  • Follow the individual patient's disease course
  • Measure change occurring during Lumizyme treatment and supportive therapies
  • Help ensure the proactive delivery of patient care
  • Evaluate treatment expectations and help healthcare providers decide on care and treatment approaches

Doctors may perform a variety of assessments, or follow-up tests, on a regular basis to monitor how Pompe disease is affecting a particular patient. For patients with Pompe disease, a general guideline of check-ups every 6 months is usually recommended. The Pompe Registry, a program sponsored by Sanofi Genzyme that tracks information about patients in order to improve understanding of Pompe disease, has developed a schedule of assessments to help guide doctors in monitoring patients. However, your doctor will determine exactly which tests, and at what frequency, are appropriate to monitor your health on ongoing basis.


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