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2. Haemophilia B - Wikipedia

   en.wikipedia.org/wiki/Haemophilia_B

   Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX. It is less common than factor VIII deficiency (haemophilia A). [3] Haemophilia B was first recognized as a distinct disease entity in 1952. [4]

3. FDA approves Pfizer's first gene therapy for rare inherited ...

   www.aol.com/news/fda-approves-pfizer-first-gene...

   The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...

4. 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 ...

5. Etranacogene dezaparvovec - Wikipedia

   en.wikipedia.org/wiki/Etranacogene_dezaparvovec

   Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. [5] [6] [7] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. [7]

6. FDA approves CSL gene therapy for hemophilia - AOL

   www.aol.com/news/fda-approves-csls-gene-therapy...

   The companies expect the gene therapy to serve as a one-time shot to cut the rate of annual bleeding for hemophilia B patients, who represent about 15% of all patients with hemophilia.

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

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

   Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally.

8. Gene therapy cures patients with hemophilia B - AOL

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

   But new research shows a one-time gene therapy could offer long-term efficacy for patients. Hemophilia B is a rare condition that prevents patients’ blood from… Gene therapy cures patients ...

9. Spark Therapeutics - Wikipedia

   en.wikipedia.org/wiki/Spark_Therapeutics

   Fidanacogene elaparvovec, previously known by its study ID number SPK-9001, [12] is a gene therapy for the treatment of hemophilia B. It was developed by Spark in partnership with Pfizer . Fidanacogene elaparvovec is an adeno-associated viral vector which is designed to transfer a working copy of the Factor IX gene into the livers of patients ...