Current treatment for Pompe Disease

Hello guys, as promised here is my second post of the week!! :D In the previous post we have discussed how Pompe is diagnosed. So what comes after diagnosis?? Its treatment! Unfortunately, there is no cure for Pompe :(( so all the medications available are used only for treatment. In the past, treatment for Pompe is mainly focused on addressing the symptoms. For example, if a patient is experiencing mobility issues due to muscle weakness, physical therapy will be used to help patients improve muscle strength. Or if the patient is experiencing breathing difficulties, mechanical ventilation is used.

However, recently this has changed. Thanks to advancement in medicine, patients can now undergo Enzyme Replacement Therapy (ERT). In ERT, a lysosomal glycogen-specific enzyme is injected into the body. The enzyme then replaces the deficient GAA enzyme and breaks down glycogen in the lysosomes. As a result, many patients start to feel better and stronger. Alglucosidase alfa is an enzyme approved for use in the U.S for ERT to treat Pompe. Alglucosidase alfa comes in 2 forms: Myozyme (used to treat infantile--onset Pompe Disease) and Lumizyme (used to treat adult-onset Pompe Disease). Although treatment are available to the public, the cost of Myozyme and Lumizyme are very costly (about $200 000-$300 000 per year). That's really alot... Fortunately, the company that created the product had conducted an Alglucosidase Alfa Temporary Access Program (ATAP) where the treatment was made available free of charge to about 200 patients. However, taking Myozyme or Lumizyme may result in side effects such as hives, diarrhoea, shortness of breath etc. which is why patients are warned before they administer the drug.

(http://www.lumizyme.com/healthcare.aspx)

Reference:
http://www.myozyme.com/
http://www.pompe.com/en/patients/managing-pompe/managing-symptoms/breathing.aspx
http://dana.org/News/Details.aspx?id=43099
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm407563.htm

Alglucosidase Alfa Temporary Access Program (ATAP)

Alglucosidase Alfa Temporary Access Program (ATAP)

Alglucosidase Alfa Temporary Access Program (ATAP)