Mucopolysaccharidoses or mukolipidose type iii Treff i utvalgte kilder: [Skjul boksen]
  1. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-us/1021/pdf/1021.pdf
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    ... hyaluronidase deficiency). Other • Pompe disease/glycogen storage disease type ... II/III, IV, Danon disease, Wolman disease, lysosomal transport defects ( ...
  2. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-gb/1021/pdf/1021.pdf
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    ... deficiency). Other • Pompe's disease/glycogen storage disease type 2 ... deficiency, mucolipidosis II/III, IV, Danon's disease, Wolman's disease, lysosomal ...
  3. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-us/1021/aetiology
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    ... hyaluronidase deficiency). Other ... Multiple sulfatase deficiency, mucolipidosis II/III, IV, Danon disease, Wolman disease, lysosomal transport defects ( ...
  4. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-gb/493/pdf/493.pdf
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    ... maple syrup urine disease types 1A and 1B, mucolipidosis IV, Niemann-Pick disease type A, and Tay-Sachs disease. Additional testing can ...
  5. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-gb/493/details
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    ... maple syrup urine disease types 1A and 1B, mucolipidosis IV, Niemann-Pick disease type A, and Tay-Sachs disease. Additional testing can ...
  6. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-us/493/details
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    ... maple syrup urine disease types 1A and 1B, mucolipidosis IV, Niemann-Pick disease type A, and Tay-Sachs disease. Additional testing can ...
  7. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-us/493/pdf/493.pdf
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    ... maple syrup urine disease types 1A and 1B, mucolipidosis IV, Niemann-Pick disease type A, and Tay-Sachs disease. Additional testing can ...
  8. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-gb/1021/management-approach
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    ... 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: ...
  9. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-us/1021/management-approach
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    ... com and vestronidase alfa has been approved for type VII (Sly syndrome). Phase III trials with vestronidase alfa are ongoing. [104] NHS; National Institute for Health Research (NIHR). NIHR Innovation Observatory ... for mucopolysaccharidosis type VII (MPS 7; Sly syndrome) NIHRIO (HSRIC) ID: ...
  10. bmj-bestpractice
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    https://bestpractice.bmj.com/topics/en-gb/1021/references
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    ... 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 ...