Glycogen storage disease type II medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Anmol Pitliya, M.B.B.S. M.D.[2]
Overview
Pharmacologic medical therapy is recommended among patients with infantile onset glycogen storage type 2 (GSD type 2). Pharmacologic medical therapies for GSD type 2 include enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase.
Medical Therapy
- Pharmacologic medical therapy is recommended among patients with infantile onset glycogen storage type 2 (GSD type 2).
- Pharmacologic medical therapies for GSD type 2 include enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase.[1]
- In 2006, ERT was approved by US Food and Drug Administration (FDA) for treatment of infantile onset GSD type 2.[2]
Glycogen storage disease type 2
- 1 Infantile onset GSD type 2
- 1.1 Enzyme replacement therapy
- Preferred regimen (1): recombinant human acid alpha-glucosidase 20 mg/Kg IV every two week
- NOTE (1): If there is poor response to initial therapy, the dose can be increased to 20 mg/kg every week.
- 1.1 Enzyme replacement therapy
References
- ↑ Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL; 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.
- ↑ "Drugs@FDA: FDA Approved Drug Products".