Skip to main content
U.S. flag

An official website of the United States government

Official websites use .gov
A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS
A lock ( ) or https:// means you’ve safely connected to the .gov website. Share sensitive information only on official, secure websites.

Because of a lapse in government funding, the information on this website may not be up to date, transactions submitted via the website may not be processed, and the agency may not be able to respond to inquiries until appropriations are enacted. The NIH Clinical Center (the research hospital of NIH) is open. For more details about its operating status, please visit cc.nih.gov. Updates regarding government operating status and resumption of normal operations can be found at opm.gov.

Search help

3,448 results

  1. 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 ...
  2. 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 ...
  3. 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 ( ...
  4. 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 ...
  5. 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, ...
  6. Appendix Data Abstraction Tables - Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117227
    ... Quality (US); 2013 Jan. (Technical Briefs, No. 12.) Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Show details Technical ... B Appendix Data Abstraction Tables Clinical Trials of Enzyme Replacement Therapy for Lysosomal Storage Diseases (MS Word, 67K) Copyright ...
  7. Summary and Implications - Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117214
    ... Quality (US); 2013 Jan. (Technical Briefs, No. 12.) Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Show details Technical ... Ratko TA, Marbella A, Godfrey S, et al. Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Rockville (MD): Agency ...
  8. Methods - Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117220
    ... Quality (US); 2013 Jan. (Technical Briefs, No. 12.) Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Show details Technical ... Ratko TA, Marbella A, Godfrey S, et al. Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Rockville (MD): Agency ...
  9. Electronic Database Search Strategies - Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117225
    ... Quality (US); 2013 Jan. (Technical Briefs, No. 12.) Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Show details Technical ... Ratko TA, Marbella A, Godfrey S, et al. Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Rockville (MD): Agency ...
  10. Summaries of Unpublished Studies - Enzyme-Replacement Therapies for Lysosomal Storage Diseases - NCBI Bookshelf 
    https://www.ncbi.nlm.nih.gov/books/NBK117222
    ... Quality (US); 2013 Jan. (Technical Briefs, No. 12.) Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Show details Technical ... Ratko TA, Marbella A, Godfrey S, et al. Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Rockville (MD): Agency ...
first · previous · 1 · 2 · 3 · 4 · 5 · 6 · 7 · 8 · 9 · 10 · next · last