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Results for search "Hemophilia".

Health News Results - 6

Trial Confirms 'Life-Changing' Impact of Gene Therapy for Hemophilia B

A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows.

People with hemophilia B saw their bleeding episodes drop by an average 71% following a single infusion of Beqvez (fidanacogene elaparvovec), researchers reported Sept. 25 in ...

FDA Approves First Gene Therapy for Severe Hemophilia A

The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder.

The treatment is not cheap: Roctavian will cost $2.9 million for a single infusion, the Associated Press reported.

"Hereditary hemophilia A is a potentially serious bleeding disorder. Severe case...

Monthly Injections Might Lower Bleeding in People With Hemophilia

An experimental injectable drug appears effective in reducing bleeds in patients with hemophilia A and B, according to a pair of new clinical trials.

Two-thirds of people with treatment-resistant hemophilia who were treated with the drug fitusiran had no bleeds at all after nine months, versus just 5% of people treated with drugs that enhance clotting, according to a trial published onlin...

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
  • |
  • 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...