Glycogen storage disease type II medical therapy: Difference between revisions

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Latest revision as of 19:41, 23 January 2018

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.

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".

Template:WS Template:WH

[pmid17151339-1] 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.

[urlDrugs@FDA:_FDA_Approved_Drug_Products-2] "Drugs@FDA: FDA Approved Drug Products".

[1]

[2]

@@ Line 1: / Line 1: @@
 __NOTOC__
 {{Glycogen storage disease type II}}
-{{CMG}}
+{{CMG}}; {{AE}} {{Anmol}}
 ==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==
-Cardiac and respiratory complications are treated symptomatically. Physical and occupational therapy may be beneficial for some patients. Alterations in diet may provide temporary improvement but will not alter the course of the disease. Genetic counseling can provide families with information regarding risk in future pregnancies.
+*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>
+*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===
-On April 28, 2006 the US Food and Drug Administration approved a biologics license application (BLA) for [[Myozyme]] (alglucosidase alfa, rhGAA), the first treatment for patients with Pompe disease primarily developed by Dr. Yuan-Tsong Chen (陳垣崇) while he was at [[Duke University]] (Dr. Chen is currently the director of the Institute of Biomedical Sciences, Academia Sinica, Taipei, Taiwan).  Myozyme falls under the FDA Orphan Drug designation and was approved under a priority review.  Myozyme is manufactured by Genzyme Corp. in Cambridge, MA, USA.  [http://www.fda.gov/bbs/topics/NEWS/2006/NEW01365.html FDA Approval News for Myozyme]
+* '''1 Infantile onset GSD type 2'''
+** '''1.1 Enzyme replacement therapy'''
-The FDA approved Myozyme for administration by intravenous infusion of solution into a vein. The safety and efficacy of Myozyme were assessed in two separate clinical trials in 39 infantile-onset patients with Pompe disease ranging in age from 1 month to 3.5 years at the time of the first infusion.
+*** 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.
-On August 14, 2006, Health Canada approved [[Myozyme]] for the treatment of Pompe disease.  On June 14, 2007 the Canadian Common Drug Review issued their recommendations regarding public funding for Myozyme therapy.  Their recommendation was to provide funding to treat a very small subset of Pompe patients (Infants less one year of age with Cardiomyopathy).<ref>[http://www.cadth.ca/media/cdr/complete/cdr_complete_Myozyme_June-14-2007_e.pdf] Canadian Common Drug Review Recommendations on Public Funding for Myozyme </ref>  The vast majority of developed countries are providing access to therapy for all diagnosed Pompe patients.<ref>[http://www.lexdon.com/article/Genzyme_Reports_Financial_Results_for/45177.html] Genzyme received broad approval in the European Union</ref>
 ==References==
 {{reflist|2}}
-{{WH}}
-{{WS}}
 [[Category:Endocrinology]]
-[[Category:Lysosomal storage diseases]]
 [[Category:Hepatology]]
+[[Category:Gastroenterology]]
+[[Category:Pediatrics]]
+[[Category:Up-To-Date]]
 [[Category:Genetic disorders]]
-[[Category:Inborn errors of metabolism]]
+[[Category:Metabolic disorders]]
+{{WS}}
+{{WH}}