The Fabrazyme shortage-A call to action for metabolic physicians

Molecular Genetics and Metabolism

Volumn 102, Issue 1, 2011, Pages 4-5

  Sirrs, Sandra ^a  

^a Adult Metabolic Diseases Clinic   (Canada)

Author keywords

Drug shortage; Enzyme replacement therapy; Fabry disease

Indexed keywords

AGALSIDASE BETA;

CLINICAL TRIAL (TOPIC); DRUG MANUFACTURE; DRUG SHORTAGE; ENZYME REPLACEMENT; HUMAN; LOW DRUG DOSE; LYSOSOME STORAGE DISEASE; NOTE; PRACTICE GUIDELINE; PRIORITY JOURNAL; RECOMMENDED DRUG DOSE; UNSPECIFIED SIDE EFFECT;

ALPHA-GALACTOSIDASE; ENZYME REPLACEMENT THERAPY; HUMANS; ISOENZYMES; PRACTICE GUIDELINES AS TOPIC;

EID: 78650889800     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2010.11.002     Document Type: Note

References (6)

1. Linthorst G.E., Germain D.P., Hollak C.E.M.., Hughes D., Rolfs A., Wanner C., Mehta A. Mehta, Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy. Mol. Genet. Metab. 2011, 102:99-102.
2. United States Food and Drug Administration Drug Safety Alerts. Available at: Last accessed November 1, 2010. http://www.fda.gov/downloads/Drugs/DrugSafety/DrugShortages/UCM187056.pdf.
3. United States Food and Drug Administration News Release Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm213212.htm. Last accessed November 1, 2010.
4. Lubanda J.C., Anijalg E., Bzduch V., Thurberg B.L., Benichou B., Tylki-Szymanksa A. Evaluation of a low dose, after a standard therapeutic dose, of agalsidase beta during enzyme replacement therapy in patients with Fabry disease. Genet. Med. 2009, 11:256-264.
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6. Grabowski G.A., Kacena K., Cole J.A., Hollak C.E.M., Zhang L., Yee J., Mistry P.K., Zimran A., Charrow J., vom Dahl S. Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1. Genet. Med. 2009, 11:92-100.