Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders—A successful strategy for clinical research of rare diseases

  1. Posset, R.
  2. Garbade, S.F.
  3. Boy, N.
  4. Burlina, A.B.
  5. Dionisi-Vici, C.
  6. Dobbelaere, D.
  7. Garcia-Cazorla, A.
  8. de Lonlay, P.
  9. Teles, E.L.
  10. Vara, R.
  11. Mew, N.A.
  12. Batshaw, M.L.
  13. Baumgartner, M.R.
  14. McCandless, S.E.
  15. Seminara, J.
  16. Summar, M.
  17. Hoffmann, G.F.
  18. Kölker, S.
  19. Burgard, P.
  20. Berry, S.A.
  21. Burrage, L.
  22. Coughlin, C.
  23. Diaz, G.A.
  24. Gallagher, R.C.
  25. Gropman, A.
  26. Harding, C.O.
  27. Lee, B.
  28. Le Mons, C.
  29. Lawrence Merritt, J.
  30. Nagamani, S.C.S.
  31. Schulze, A.
  32. Stricker, T.
  33. Tuchman, M.
  34. Waisbren, S.
  35. WeisfeldAdams, J.
  36. Wong, D.
  37. Yudkoff, M.
  38. Arnoux, J.
  39. Barić, I.
  40. Bosch, A.M.
  41. Chabrol, B.
  42. Chakrapani, A.
  43. CortèsSaladefont, E.
  44. Couce, M.L.
  45. Eyskens, F.
  46. de Laet, C.
  47. de Meirleir, L.
  48. Freisinger, P.
  49. Gleich, F.
  50. Grünewald, S.
  51. Häberle, J.
  52. Hwu, W.
  53. Jalan, A.
  54. Karall, D.
  55. Lindner, M.
  56. Lund, A.M.
  57. Martinelli, D.
  58. Murphy, E.
  59. Mühlhausen, C.
  60. Olivieri, G.
  61. Ottolenghi, C.
  62. Rodrigues, E.
  63. Rubert, L.
  64. Sarajlija, A.
  65. Schiff, M.
  66. Sokal, E.
  67. SykutCegielska, J.
  68. Walter, J.H.
  69. Williams, M.
  70. Zeman, J.
  71. Show all authors +
Journal:
Journal of Inherited Metabolic Disease

ISSN: 1573-2665 0141-8955

Year of publication: 2019

Volume: 42

Issue: 1

Pages: 93-106

Type: Article

DOI: 10.1002/JIMD.12031 GOOGLE SCHOLAR