December 21st 2023
Men with severe or moderately severe hemophilia B continue to display low bleeding rates and very little use of replacement therapies for at least three years following a single dose of the approved gene therapy hemgenix.
Gene Therapies for Hemophilia. Possibly Curative, Likely Ultraexpensive
September 8th 2022Three gene therapies in late-stage development are designed to supply working copies of genes that people with hemophilia are missing. They may be one-time, curative treatments, but the seven-figure price tags will be an issue.
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