Alglucosidase alfa (Lumizyme, Myozyme, Genzyme) is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease (Glycogen storage disease type II), a rare lysosomal storage disorder (LSD). Chemically speaking, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase. It is the first drug available to treat this disease.
Orphan drug pharmaceutical company, Genzyme, markets alglucosidase alfa as "Myozyme". In 2006, the U.S. Food and Drug Administration (FDA) approved Myozyme as a suitable ERT treatment for children. Some health plans have refused to subsidize Myozyme for adult patients because it lacks approval for treatment in adults, as well as its high cost (US$300,000/yr for life).
Common observed adverse reactions to alglucosidase alfa treatment are pneumonia, respiratory complications, infections and fever. More serious reactions reported include heart and lung failure and allergic shock. Myozyme boxes carry warnings regarding the possibility of life-threatening allergic response.
- Kishnani PS; Corzo D; Nicolino M; et al. (2007). "Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease". Neurology. 68 (2): 99–109. doi:10.1212/01.wnl.0000251268.41188.04. PMID 17151339. Unknown parameter
- "FDA Approves First Treatment for Pompe Disease" (Press release). FDA. 2006-04-28. Retrieved 2008-07-07.
- Geeta Anand (2007-09-18). "As Costs Rise, New Medicines Face Pushback". Wall Street Journal. Dow Jones & Company. Retrieved 2008-07-07.
- Myozyme (alglucosidase alfa), Genzyme official website
- MTAP (Myozyme Temporary Access Program), Genzyme official website
World's largest food, health, weight loss, wellnesspedia and encyclopedia
If you are a medical professional or an expert in any field of medicine, please join us in building the world's largest weight loss and wellness encyclopedia created by experts in the field, not by the crowd. WikiMD is sponsored by W8MD weight loss, sleep and medical aesthetic centers
W8MD Weight Loss, Sleep & Medical Aesthetic Centers
Since its inception in 2011, W8MD’s insurance physician weight loss program has successfully helped thousands of patients succeed in not only losing weight but also keep it off with an ongoing maintenance plan.
FANTASTIC PROGRAM. TRULY A LIFE CHANGER. D.M. LOST 100 LBS^^
^^Individual results may vary.
W8MD weight loss, sleep and medical spa blogs
Support our sponsors
W8MD weight loss, sleep and medspa centers sponsors WikiMD.
W8MD's Locations for losing weight, sleeping better and looking your best
King of Prussia
Other W8MD blogs