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Trial Confirms 'Life-Changing' Impact of Gene Therapy for Hemophilia B
- Dennis Thompson HealthDay Reporter
- September 26, 2024
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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...
FDA Approves First Gene Therapy for Severe Hemophilia A
- Cara Murez HealthDay Reporter
- June 30, 2023
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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
Monthly Injections Might Lower Bleeding in People With Hemophilia
- Dennis Thompson HealthDay Reporter
- March 31, 2023
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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 af...
A Gene Therapy for Hemophilia That Costs $3.5 Million Gets FDA Approval
- Cara Murez HealthDay Reporter
- November 23, 2022
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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.
Gene Therapy Makes Inroads Against a Form of Hemophilia
- By Dennis Thompson HealthDay Reporter
- July 22, 2022
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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 ...
Gene Therapy Could Be Big Advance Against Hemophilia
- Robert Preidt
- November 18, 2021
- Página completa
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 mis...