Remove tag bone-marrow-transplant
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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

A bone marrow transplant from a donor can offer a potential cure, but it is often difficult to find a matching donor and the procedure carries the risk of rejection by the immune system. It can also make patients more susceptible to complications like anemia, jaundice, infections and gallstones.

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bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy

pharmaphorum

But haematopoietic stem cell transplantation is only possible when a donor with a matching human leukocyte antigen signature, within the correct age range, is available. million in Europe.