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The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the majority of whom are...
Federal officials Friday approved two gene therapy treatments for sickle cell disease, welcome news for thousands with the inherited blood disorder that causes terrible pain crises and...
The two gene therapies are the first approved in the U.S. for sickle cell disease. The FDA has previously OK’d 15 gene therapies for other conditions.
The US Food and Drug Administration (FDA) approved lovotibeglogene autotemcel in December 2023.
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).
In November 2019, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease for those twelve years of age and older. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.
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