Enzyme replacement and substrate reduction therapy for Gaucher disease

Background Gaucher disease, a rare disorder, is caused by inherited deficiency of the enzyme glucocerebrosidase. It is unique among the ultra‐orphan disorders in that four treatments are currently approved by various regulatory authorities for use in routine clinical practice. Hitherto, because of t...

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Veröffentlicht in:	Cochrane database of systematic reviews 2015-03, Vol.2015 (4), p.CD010324
Hauptverfasser:	Shemesh, Elad, Deroma, Laura, Bembi, Bruno, Deegan, Patrick, Hollak, Carla, Weinreb, Neal J, Cox, Timothy M
Format:	Artikel
Sprache:	eng
Schlagworte:	1-Deoxynojirimycin - adverse effects 1-Deoxynojirimycin - analogs & derivatives 1‐Deoxynojirimycin Blood disorders Child health Enzyme Inhibitors Enzyme Inhibitors - adverse effects Enzyme Replacement Therapy Enzyme Replacement Therapy - methods Gaucher Disease Gaucher Disease - blood Gaucher Disease - drug therapy Genetic disorders Glucosylceramidase Glucosylceramidase - therapeutic use Hemoglobin A Hemoglobin A - metabolism Hepatomegaly Hepatomegaly - drug therapy Humans INBORN ERRORS OF METABOLISM Medicine General & Introductory Medical Sciences Platelet Count Randomized Controlled Trials as Topic Splenomegaly Splenomegaly - drug therapy Substrate Specificity TREATMENT
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