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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.

The U.S. Food and Drug Administration approved the new gene therapy Hemgenix on Nov. 22. Soon after, drugmaker CSL Behring revealed its cost.

The company said its drug would ultimately reduce health care costs because patients with the genetic disorder would ne...

Gene Therapy Makes Inroads Against a Form of Hemophilia

People with hemophilia B could find their bleeding risk dramatically reduced with just one injection of an experimental gene therapy, a new study reports.

Hemophilia B is a rare and inherited genetic disorder in which people have low levels of the

  • By Dennis Thompson HealthDay Reporter
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  • July 22, 2022
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  • Full Page
  • Gene Therapy Could Be Big Advance Against Hemophilia

    Gene therapy shows promise in reducing, and even halting, potentially life-threatening bleeding events in people with hemophilia, researchers report.

    Hemophilia A is the most common inherited bleeding disorder, affecting one in 5,000 males worldwide. It's caused by a missing coagulation factor called FVIII.

    The current standard of care involves regular infusions of the FVIII protein...

    Early Trial Offers New Hope for People With Hemophilia

    Researchers may have found a way for people with severe hemophilia to take their standard treatment less often, if the results of an early trial pan out.

    In what experts called a feat of bioengineering, scientists were able to create a "fusion protein" that may extend the interval between treatments for hemophilia -- from about every couple of days to once a week.

    The early ...

    Gene Therapy May Be Long-Term Cure for Type of Hemophilia

    A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate.

    Patients who received the one-time intravenous therapy continue to have a more than 90% decrease in bleeding events two to three years after their initial treatment, researchers reported Jan. 1 in the New England Journal of Medicine.

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