Glycogen storage disease type II medical therapy: Difference between revisions
No edit summary |
No edit summary |
||
Line 5: | Line 5: | ||
==Overview== | ==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== | ==Medical Therapy== | ||
*Pharmacologic medical therapy is recommended among patients with infantile onset glycogen storage type 2 (GSD type 2). | *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.<ref name="pmid17151339">{{cite journal| author=Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL et al.| title=Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. | journal=Neurology | year= 2007 | volume= 68 | issue= 2 | pages= 99-109 | pmid=17151339 | doi=10.1212/01.wnl.0000251268.41188.04 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=17151339 }}</ref> | *Pharmacologic medical therapies for GSD type 2 include [[enzyme replacement therapy]] (ERT) with recombinant human [[acid alpha-glucosidase]].<ref name="pmid17151339">{{cite journal| author=Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL et al.| title=Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. | journal=Neurology | year= 2007 | volume= 68 | issue= 2 | pages= 99-109 | pmid=17151339 | doi=10.1212/01.wnl.0000251268.41188.04 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=17151339 }}</ref> | ||
*In 2006, ERT was approved by US Food and Drug Administration (FDA) for treatment of infantile onset GSD type 2.<ref name="urlDrugs@FDA: FDA Approved Drug Products">{{cite web |url=https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=BasicSearch.process |title=Drugs@FDA: FDA Approved Drug Products |format= |work= |accessdate=}}</ref> | *In 2006, ERT was approved by [[US Food and Drug Administration]] ([[FDA]]) for treatment of infantile onset GSD type 2.<ref name="urlDrugs@FDA: FDA Approved Drug Products">{{cite web |url=https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=BasicSearch.process |title=Drugs@FDA: FDA Approved Drug Products |format= |work= |accessdate=}}</ref> | ||
===Glycogen storage disease type 2=== | ===Glycogen storage disease type 2=== | ||
* '''1 Infantile onset GSD type 2''' | * '''1 Infantile onset GSD type 2''' | ||
** '''1.1 Enzyme replacement therapy''' | ** '''1.1 Enzyme replacement therapy''' | ||
*** Preferred regimen (1): recombinant human acid alpha-glucosidase 20 mg/Kg IV every two week | *** 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 | **: '''NOTE (1):''' If there is poor response to initial therapy, the dose can be increased to 20 mg/kg every week. | ||
==References== | ==References== | ||
{{reflist|2}} | {{reflist|2}} |
Revision as of 20:05, 22 January 2018
Glycogen storage disease type II Microchapters |
Differentiating Glycogen storage disease type II from other Diseases |
---|
Diagnosis |
Treatment |
Case Studies |
Glycogen storage disease type II medical therapy On the Web |
American Roentgen Ray Society Images of Glycogen storage disease type II medical therapy |
Glycogen storage disease type II medical therapy in the news |
Directions to Hospitals Treating Glycogen storage disease type II |
Risk calculators and risk factors for Glycogen storage disease type II medical therapy |
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".