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Hyperhomocysteinemia in patients with acute porphyrias: a possible effect of ALAS1 modulation by siRNAm therapy and its control by vitamin supplementation

  • Andrea Ricci
    Affiliations
    Internal Medicine Unit, Department of Medical and Surgical Science for Children and Adults,University of Modena e Reggio Emilia
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  • Matteo Marcacci
    Affiliations
    Internal Medicine Unit, Department of Medical and Surgical Science for Children and Adults,University of Modena e Reggio Emilia
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  • Chiara Cuoghi
    Affiliations
    Internal Medicine Unit, Department of Medical and Surgical Science for Children and Adults,University of Modena e Reggio Emilia
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  • Antonello Pietrangelo
    Affiliations
    Internal Medicine Unit, Department of Medical and Surgical Science for Children and Adults,University of Modena e Reggio Emilia
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  • Paolo Ventura
    Correspondence
    Corresponding author at: Internal Medicine Unit, Department of Medical and Surgical Science for Children and Adults, Regional Reference Centre for Diagnosing and Management of Porphyrias, University of Modena and Reggio Emilia, Azienda Ospedaliero-Universitaria Policlinico of Modena, Largo del Pozzo 71, 41124 Modena, Italy.
    Affiliations
    Internal Medicine Unit, Department of Medical and Surgical Science for Children and Adults,University of Modena e Reggio Emilia
    Search for articles by this author

      Highlights

      • ALAS1 siRNAm Givosiran is innovative and effective treatment in patients with AHPs.
      • During Givosiran treatment patients show an increase in plasma homocysteine.
      • Moderate to severe hyperhomocysteinaemia may be risk factor for thrombosis.
      • Vitamin supplementation significantly reduces Givosiran-induced hyperhomocysteinaemia.

      Keywords

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