The treatment is the enzyme alglucosidase alfa (also known as Myozyme). It is manufactured by Sanofi Genzyme. Myozyme is used to treat adults, children and adolescents of all ages who have a confirmed diagnosis of Pompe disease. Myozyme can replace the natural enzyme which is lacking in Pompe disease.
You can find out more about Myozyme by downloading the file called "Patient Information" on the bottom of this page.
For more information about treatment for Pompe disease, you should consult your doctor.
In case you experience any adverse event during any treatment for Pompe disease, please contact your treating physician immediately.
Pompe disease can affect many different parts of the body. Because of this, management of Pompe disease needs input from a wide range of medical specialists and supportive-care health professionals.
Doctors from various medical areas may be involved in managing the care of a person with Pompe disease. Some doctors have expertise in managing more than one aspect of the condition, whilst others focus on one particular area:
- Neuromuscular specialists and neurologists
Experts in muscular diseases and related loss of muscle function (one of the main signs of Pompe disease)
- Geneticists and metabolic specialists
Experts in diseases passed through families and problems involving chemical processes within living cells
- Respiratory specialists
Experts in conditions affecting the lungs, resulting in breathing problems (one of the main signs of Pompe disease)
Experts in heart conditions (especially relevant for infants with Pompe disease)
Experts in disorders of the digestive tract (i.e. the stomach and guts)
Supportive-care health professionals
Once diagnosed with Pompe disease, a person may see many different healthcare professionals for supportive care, to help manage their condition and live life as normally as possible: