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2. Etranacogene dezaparvovec - Wikipedia

   en.wikipedia.org/wiki/Etranacogene_dezaparvovec

   The safety and effectiveness of etranacogene dezaparvovec were evaluated by the US Food and Drug Administration (FDA) in two studies of 57 adult men 18 to 75 years of age with severe or moderately severe hemophilia B. [7] Effectiveness was established based on decreases in the men's annualized bleeding rate (ABR). [7]

3. Fidanacogene elaparvovec - Wikipedia

   en.wikipedia.org/wiki/Fidanacogene_elaparvovec

   In the US, fidanacogene elaparvovec is indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy; or have current or historical life-threatening hemorrhage; or have repeated, serious spontaneous bleeding episodes; and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var ...

4. List of drugs granted breakthrough therapy designation

   en.wikipedia.org/wiki/List_of_drugs_granted...

   Treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who are receiving routine prophylaxis and without pre-existing neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid detected by an FDA-approved test. [3] Inavolisib: Genentech

5. FDA Approves Pfizer's Second Hemophilia Drug With Six Months

   www.aol.com/fda-approves-pfizers-second...

   Beqvez is a one-time treatment designed to enable hemophilia B patients to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that ...

6. Gene therapy cures patients with hemophilia B - AOL

   www.aol.com/gene-therapy-cures-patients...

   Story at a glance Hemophilia B occurs when patients lack sufficient levels of the blood protein factor IX. Some current treatments for the condition involve repeated infusions of the protein. But ...

7. Biogen Idec Says Long-Lasting Hemophilia Drug Meets ... - AOL

   www.aol.com/news/2010-07-12-biogen-idec...

   Biogen Idec (BIIB) and Swedish Orphan Biovitrum said a potential hemophilia B treatment has met safety goals in an early-stage study. The study involved 14 previously treated patients, and the ...