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Hemophagocytic lymphohistiocytosis following gene replacement therapy in a child with type 1 spinal muscular atrophy.

Francesca GallettaUgo CucinottaLucia MarsegliaAnnalisa CacciolaRomina GallizziSalvatore CuzzocreaSonia MessinaAntonio ToscanoEloisa Gitto
Published in: Journal of clinical pharmacy and therapeutics (2022)
To our knowledge, this is the first case of HLH following gene replacement therapy with OA, described in literature.
Keyphrases
  • replacement therapy
  • smoking cessation
  • copy number
  • genome wide
  • systematic review
  • healthcare
  • genome wide identification
  • mental health
  • dna methylation
  • knee osteoarthritis
  • gene expression
  • transcription factor