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The FDA is reviewing a drug that could cure sickle cell anemia. Right now bone marrow or stem cell transplants are the only FDA-approved cures.
It was the first approved drug for the treatment of sickle cell anaemia, and was shown to decrease the number and severity of attacks in 1995 [110] and shown to possibly increase survival time in a study in 2003. [111] This is achieved, in part, by reactivating fetal haemoglobin production in place of the haemoglobin S that causes sickle cell ...
The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era ...
Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). [ 64 ] The gene therapy is made from the recipient's own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a ...
The FDA is expected to decide on the second gene therapy for sickle cell, made by Bluebird Bio, before the end of the year. Bluebird's treatment works differently.
The FDA said treatment for severe sickle cell is an “unmet medical need.” When someone has sickle cell disease their red blood cells don’t function the way they should. Red blood cells are ...
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