Results 1-7 of about 12|
  1. ... hyaluronidase deficiency). Other • Pompe disease/glycogen storage disease type ... II/III, IV, Danon disease, Wolman disease, lysosomal transport defects ( ...
    Source: BMJ Best Practice
    Information Type: Oppslagsverk
  2. ... deficiency). Other • Pompe's disease/glycogen storage disease type 2 ... deficiency, mucolipidosis II/III, IV, Danon's disease, Wolman's disease, lysosomal ...
    Source: BMJ Best Practice
    Information Type: Oppslagsverk
  3. ... hyaluronidase deficiency). Other ... Multiple sulfatase deficiency, mucolipidosis II/III, IV, Danon disease, Wolman disease, lysosomal transport defects ( ...
    Source: BMJ Best Practice
    Information Type: Oppslagsverk
  4. ... maple syrup urine disease types 1A and 1B, mucolipidosis IV, Niemann-Pick disease type A, and Tay-Sachs disease. Additional testing can ...
    Source: BMJ Best Practice
    Information Type: Oppslagsverk
  5. ... maple syrup urine disease types 1A and 1B, mucolipidosis IV, Niemann-Pick disease type A, and Tay-Sachs disease. Additional testing can ...
    Source: BMJ Best Practice
    Information Type: Oppslagsverk
  6. ... com and vestronidase alfa has been approved for type VII (Sly syndrome). Phase III trials with vestronidase alfa are ongoing. [97] NHS; National Institute for Health Research (NIHR). NIHR Innovation Observatory ... for mucopolysaccharidosis type VII (MPS 7; Sly syndrome) NIHRIO (HSRIC) ID: ...
    Source: BMJ Best Practice
    Information Type: Oppslagsverk
  7. ... with Gaucher disease: long-term data from phase III clinical trials. Am J Hematol. 2015 ... in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial. JAMA. 2015 ...
    Source: BMJ Best Practice
    Information Type: Oppslagsverk