Acta Paediatrica

Cover image for Vol. 97 Issue s457

Special Issue: Natural course, CNS pathophysiology and next targets

April 2008

Volume 97, Issue Supplement s457

Pages 1–122

  1. Natural course, CNS pathophysiology and next targets

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Sponsored by Shire Human Genetic Therapies a business unit of Shire Pharmaceuticals Ltd

      Foreword
      Seventh International Symposium on Lysosomal Storage Diseases (pages 1–2)

      Johannes Aerts, Michael Beck, Rick Martin and Maurizio Scarpa

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00647.x

  2. Commentary

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Lysosomal storage diseases: new challenges (pages 5–6)

      Maurizio Scarpa and Yoshikatsu Eto

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00645.x

  3. Reviews

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Biomarkers for lysosomal storage disorders: identification and application as exemplified by chitotriosidase in Gaucher disease (pages 7–14)

      Johannes M Aerts, Marielle J Van Breemen, Anton P Bussink, Karen Ghauharali, Richard Sprenger, Rolf G Boot, Johanna E Groener, Carla E Hollak, Mario Maas, Suzanne Smit, Huub C Hoefsloot, Age K Smilde, Johannes PC Vissers, Sheryas De Jong, Dave Speijer and Chris G De Koster

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2007.00641.x

    2. CNS-directed gene therapy for lysosomal storage diseases (pages 22–27)

      Mark S Sands and Mark E Haskins

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00660.x

  4. Commentary

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Fabry disease: beyond men (pages 31–32)

      Atul Mehta and Rossella Parini

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00642.x

  5. Reviews

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Newborn screening for lysosomal storage disorders (pages 33–37)

      Dietrich Matern

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00652.x

    2. Early therapeutic intervention in females with Fabry disease? (pages 41–47)

      Derralynn A Hughes

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00649.x

  6. Original Articles

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Apoptotic abnormalities in differential gene expression in peripheral blood mononuclear cells from children with Fabry disease (pages 48–52)

      David F Moore, Ehud Goldin, Monique P Gelderman, Chevalia Robinson, Jessica Baer, Markus Ries, Abdel Elkahloun, Roscoe O Brady and Raphael Schiffmann

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00654.x

  7. Commentary

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Hunter syndrome: to treat or not to treat (pages 55–56)

      Joseph Muenzer and Ana Maria Martins

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00644.x

  8. Reviews

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Magnetic resonance imaging findings in Hunter syndrome (pages 61–68)

      Chelsea T Finn, Leonardo Vedolin, Ida V Schwartz, Roberto Giugliani, Charlotte A Haws, Andrew P Prescot and Perry F Renshaw

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00646.x

    2. Methods for assessing neurodevelopment in lysosomal storage diseases and related disorders: a multidisciplinary perspective (pages 69–75)

      Holly R Martin, Michele D Poe, Debra Reinhartsen, Rebecca E Pretzel, Jackson Roush, Angela Rosenberg, Stacey C Dusing and Maria L Escolar

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00651.x

  9. Commentary

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Gaucher disease: improving management (pages 81–82)

      Bruno Bembi and Patrick Deegan

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00643.x

  10. Reviews

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. Gaucher disease: unmet treatment needs (pages 83–87)

      Atul Mehta

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00653.x

    2. Substrate reduction therapy (pages 88–93)

      Frances M Platt and Mylvaganam Jeyakumar

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00656.x

  11. POSTER ABSTRACTS

    1. Top of page
    2. Natural course, CNS pathophysiology and next targets
    3. Commentary
    4. Reviews
    5. Commentary
    6. Reviews
    7. Original Articles
    8. Commentary
    9. Reviews
    10. Commentary
    11. Reviews
    12. POSTER ABSTRACTS
    1. MUCOPOLYSACCHARIDOSIS DISORDERS (pages 97–105)

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00657_1.x

    2. FABRY DISEASE (pages 106–113)

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00657_2.x

    3. GAUCHER DISEASE (pages 114–118)

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00657_3.x

    4. OTHER LYSOSOMAL STORAGE DISEASES (pages 119–122)

      Version of Record online: 13 MAR 2008 | DOI: 10.1111/j.1651-2227.2008.00657_4.x

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