Cario, Holger ORCID: 0000-0002-6923-488X and Lobitz, Stephan ORCID: 0000-0001-5398-0610 (2019). What's new in diagnostics and treatment of hemoglobinopathies? Dtsch. Med. Wochenschr., 144 (11). S. 719 - 724. STUTTGART: GEORG THIEME VERLAG KG. ISSN 1439-4413

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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 pathophysiology of vaso-occlusive complications e.g. by inhibiting selectins are currently thoroughly investigated and some of them will probably get approval very soon.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Cario, HolgerUNSPECIFIEDorcid.org/0000-0002-6923-488XUNSPECIFIED
Lobitz, StephanUNSPECIFIEDorcid.org/0000-0001-5398-0610UNSPECIFIED
URN: urn:nbn:de:hbz:38-138563
DOI: 10.1055/a-0601-4921
Journal or Publication Title: Dtsch. Med. Wochenschr.
Volume: 144
Number: 11
Page Range: S. 719 - 724
Date: 2019
Publisher: GEORG THIEME VERLAG KG
Place of Publication: STUTTGART
ISSN: 1439-4413
Language: German
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
Medicine, General & InternalMultiple languages
Refereed: Yes
URI: http://kups.ub.uni-koeln.de/id/eprint/13856

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