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Goal attainment in PMM2-CDG: A new approach measuring meaningful clinical outcomes

  • Sanne Verberkmoes
  • , Gina L. Mazza
  • , Andrew C. Edmondson
  • , Fernando Scaglia
  • , Seishu Horikoshi
  • , Bryce Kuschel
  • , Mirian C.H. Janssen
  • , Jehan Mousa
  • , Austin Larson
  • , Rameen Shah
  • , Georgia McDonald
  • , Kyriaki Sarafoglou
  • , Gerard Berry
  • , Tamas Kozicz
  • , Christina Lam
  • , Eva Morava

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Biochemistry, Genetics and Molecular Biology