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  1. MedlinePlus Drug Information: Miglustat 
    https://medlineplus.gov/druginfo/meds/a604015.html
    ... and who have not been improving on their enzyme therapy. Miglustat is in a class of medications called enzyme inhibitors. It works by preventing the body from ...
  2. MedlinePlus Drug Information: Cipaglucosidase alfa-atga 
    https://medlineplus.gov/druginfo/meds/a623057.html
    ... and who have not been improving on their enzyme therapy. Cipaglucosidase alfa-atga is in a class of medications called recombinant lysosomal enzymes. It works by helping break glycogen down to ...
  3. Background - Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117221
    ... Quality (US); 2013 Jan. (Technical Briefs, No. 12.) Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Show details Technical ... strong. 4 Therapeutic Measures for Lysosomal Storage Diseases Enzyme-Replacement Therapy Within the United States, the term “enzyme replacement ...
  4. Resource Bibliography - Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117228
    ... Quality (US); 2013 Jan. (Technical Briefs, No. 12.) Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Show details Technical ... Tesmoingt C, Lidove O, Reberga A, et al. Enzyme therapy in Fabry disease: severe adverse events associated with ...
  5. Findings - Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117223
    ... Quality (US); 2013 Jan. (Technical Briefs, No. 12.) Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Show details Technical ... Next > Findings Guiding Question 1. What FDA-approved enzyme-replacement therapy (ERT) products are available for lysosomal storage diseases ( ...
  6. Appendix References - Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117215
    ... Quality (US); 2013 Jan. (Technical Briefs, No. 12.) Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Show details Technical ... Grabowski GA, Barton NW, Pastores G, et al. Enzyme therapy in type 1 Gaucher disease: comparative efficacy of ...
  7. Enzyme Replacement Therapy - LiverTox - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK548796
    ... Diseases ; 2012-. Drug Records Search term < Prev Next > Enzyme Replacement Therapy Last Update: March 10, 2016 . OVERVIEW Introduction Enzyme ... volume and fat content improved more with active enzyme therapy than placebo; one patient had a mild hypersensitivity ...
  8. The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease: a systematic ... 
    https://www.ncbi.nlm.nih.gov/books/NBK62295
    ... Next > The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease: a systematic review M ... of Leicester, UK * Corresponding author Published: 2006 . Objective Enzyme replacement therapy (ERT; intravenous imiglucerase) is used in the treatment ...
  9. Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117219
    ... of Medicine, National Institutes of Health. < Prev Next > Enzyme-Replacement Therapies for Lysosomal Storage Diseases Technical Briefs, No. 12 ... systems involved, and the degree of impairment. However, enzyme-replacement therapy (ERT) is now commercially available for six LSDs, ...
  10. A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's ... 
    https://www.ncbi.nlm.nih.gov/books/NBK62284
    ... of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1 ... and cost-effectiveness of the administration of intravenous enzyme replacement therapy (ERT) to symptomatic patients for the prevention of ...
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