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Spark's investigational hemophilia A gene therapy, administered via a one-time intravenous infusion, is designed to trigger the production of therapeutic levels of factor VIII via the liver cells.
Christi Humphrey, LCSW of Hemophilia of Georgia and Jhon Velasco, MA of NHF discuss the principles of anger and how it works in/through the body.
NHF has learned of the recent death of a patient who had been participating in a gene therapy clinical trial for a rare muscle disorder known as X-Linked Myotubular Myopathy.
Pending regulatory clearance, CSL Behring would acquire exclusive global license rights from uniQure to commercialize etranacogene dezaparvovec, otherwise known as AMT-061.
The factor product donation made by the two companies is in support of the World Federation of Hemophilia's Humanitarian Aid Program.