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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.

Anger Happens: Coping During COVID-19

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.

Two Deaths Reported in AAV-Based Gene Therapy Trials for Rare Muscle Disorder

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.

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