How I treat ADA deficiency.

  title={How I treat ADA deficiency.},
  author={H Bobby Gaspar and Alessandro Aiuti and Fulvio Porta and Fabio Candotti and Michael S. Hershfield and Luigi Daniele Notarangelo},
  volume={114 17},
Adenosine deaminase deficiency is a disorder of purine metabolism leading to severe combined immunodeficiency (ADA-SCID). Without treatment, the condition is fatal and requires early intervention. Haematopoietic stem cell transplantation is the major treatment for ADA-SCID, although survival following different donor sources varies considerably. Unlike other SCID forms, 2 other options are available for ADA-SCID: enzyme replacement therapy (ERT) with pegylated bovine ADA, and autologous… 

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