A gene therapy for hemophilia that costs $3.5 million gets FDA approval
People with one form of the genetic blood disorder hemophilia now have a one-time treatment with a $3.5 million price tag.
Nov 23, 2022
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Hemic and Lymphatic Diseases: News and Research on Hemophilia B
MeSH tree: C15.378.100.100.510
Gene therapy with AAV serotype 2 capsid variants for improved liver disease treatments
There are numerous severe, difficult-to-treat monogenetic diseases that are caused by a defect in a single gene—also in the liver. These include the blood coagulation disorders hemophilia A or B or the metabolic disease phenylketonuria. ...
Oct 26, 2022
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Novel gene therapy could reduce bleeding risk for hemophilia patients
A single gene therapy injection could dramatically reduce the bleeding risk faced by people with hemophilia B, finds a study involving UCL researchers.
Jul 20, 2022
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