What's new in diagnostics and treatment of hemoglobinopathies?

  • Cario, Holger
  • Lobitz, Stephan
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Publication date
January 2019
Publisher
Georg Thieme Verlag KG

Abstract

Improvement of care for thalassemia during previous decades focussed on the development of new diagnostic and treatment modalities concerning secondary hemochromatosis resulting from symptomatic treatment with regular transfusion. In addition, hematopoetic stem transplantation as the so far only curative approach had been developed and constantly improved. New approaches for the treatment of thalassemias already entering clinical practice include e.g. additive gene therapy and medical treatment with new drugs like luspatercept. For sickle cell disease, a variety of treatment approaches either to correct the genetic change by gene therapy, to affect the underlying mechanism of sickling e.g. by R-state- stabilising agents, or later the pathop...

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