Clinical trial updates

Alpha-thalassaemia major

 

 

Update: 05 November 2020

  • Researchers at the University of California – San Francisco (UCSF) are conducting a Phase I clinical trial to test a new strategy for conducting in utero stem cell transplantation to foetuses diagnosed with alpha thalassaemia using the mother’s stem cells at the same time as a fetal blood transfusion.
  • Foetuses with alpha thalassaemia major do not survive unless in utero transfusions are provided, furthermore posing many challenges to the mother’s health also.
  • Already two patients have been treated in this way and are now thriving toddlers.
  • More information is available at https://thalassaemia.org.cy/education/learn-about-thalassaemia/haemoglobin-disorders/alpha-thalassaemia/

Sources:

https://fetus.ucsf.edu/research/intrauterine-therapy-alpha-thalassemia-major

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