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Title: :  Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A
PaperId: :  20344
Published in:   International Journal Of Advance Research And Innovative Ideas In Education
Publisher:   IJARIIE
e-ISSN:   2395-4396
Volume/Issue:    Volume 9 Issue 3 2023
DUI:    16.0415/IJARIIE-20344
Licence: :   IJARIIE is licensed under a Creative Commons Attribution-ShareAlike 4.0 International License.

Author NameAuthor Institute
Sanaullah SafiCheragh Medical Institute of Higher Education
Hedayaturahman HabibzaiMili Institute of Higher Education
sayed tariq PachakhanSpinghar Medical Institute

Abstract

life Sciences
Hemophilia A, Gene Therapy, FIII, Valoctocogene Roxaparvovec (Roctavian), Adeno-Associated Virus (AAV), BioMarin Pharmaceutical Inc
Hemophilia is a hemorrhagic disease characterized by the absence or deficiency of clotting factor VIII (hemophilia A) or clotting factor IX (hemophilia B) and hemophilia C or factor XI deficiency. It affects 1 in 5,000 living males, 1 in 40,000 live males, and 1 in 100,000 live births worldwide (Hemophilia A, B, and C). Emicizumab is a monoclonal antibody that bridges activated factors IX and X to perform the duties of activated factor VIII when it is absent, restoring hemostasis. Intravenous infusion of exogenous FVIII is the current gold standard in hemophilia A treatment. Prophylactic coagulation factor replacement has been shown to reduce arthropathy, decrease the frequency of bleeds, and improve quality of life. Gene therapy for hemophilia involves targeting hepatocytes, allowing for long-term expression of defective FVIII while maintaining stable plasma FVIII concentrations. The first gene therapy for hemophilia A is Roctavian. Valoctocogene roxaparvovec, the active ingredient in Roctavian, is based on an adeno-associated virus (AAV) that has been altered not to infect people. Roxaparvovec was granted conditional marketing authorization in the EU to treat severe hemophilia A in adults without FVIII inhibitors and AAV5 antibodies.

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IJARIIE Sanaullah Safi, Hedayaturahman Habibzai, and sayed tariq Pachakhan. "Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A" International Journal Of Advance Research And Innovative Ideas In Education Volume 9 Issue 3 2023 Page 2150-2157
MLA Sanaullah Safi, Hedayaturahman Habibzai, and sayed tariq Pachakhan. "Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A." International Journal Of Advance Research And Innovative Ideas In Education 9.3(2023) : 2150-2157.
APA Sanaullah Safi, Hedayaturahman Habibzai, & sayed tariq Pachakhan. (2023). Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A. International Journal Of Advance Research And Innovative Ideas In Education, 9(3), 2150-2157.
Chicago Sanaullah Safi, Hedayaturahman Habibzai, and sayed tariq Pachakhan. "Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A." International Journal Of Advance Research And Innovative Ideas In Education 9, no. 3 (2023) : 2150-2157.
Oxford Sanaullah Safi, Hedayaturahman Habibzai, and sayed tariq Pachakhan. 'Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A', International Journal Of Advance Research And Innovative Ideas In Education, vol. 9, no. 3, 2023, p. 2150-2157. Available from IJARIIE, https://ijariie.com/AdminUploadPdf/Understanding_of_Valoctocogene_Roxaparvovec__Roctavian__as_a_Breakthrough_Gene_Therapy_for_Hemophilia_Type_A_ijariie20344.pdf (Accessed : 06 December 2024).
Harvard Sanaullah Safi, Hedayaturahman Habibzai, and sayed tariq Pachakhan. (2023) 'Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A', International Journal Of Advance Research And Innovative Ideas In Education, 9(3), pp. 2150-2157IJARIIE [Online]. Available at: https://ijariie.com/AdminUploadPdf/Understanding_of_Valoctocogene_Roxaparvovec__Roctavian__as_a_Breakthrough_Gene_Therapy_for_Hemophilia_Type_A_ijariie20344.pdf (Accessed : 06 December 2024)
IEEE Sanaullah Safi, Hedayaturahman Habibzai, and sayed tariq Pachakhan, "Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A," International Journal Of Advance Research And Innovative Ideas In Education, vol. 9, no. 3, pp. 2150-2157, May-Jun 2023. [Online]. Available: https://ijariie.com/AdminUploadPdf/Understanding_of_Valoctocogene_Roxaparvovec__Roctavian__as_a_Breakthrough_Gene_Therapy_for_Hemophilia_Type_A_ijariie20344.pdf [Accessed : 06 December 2024].
Turabian Sanaullah Safi, Hedayaturahman Habibzai, and sayed tariq Pachakhan. "Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A." International Journal Of Advance Research And Innovative Ideas In Education [Online]. volume 9 number 3 (06 December 2024).
Vancouver Sanaullah Safi, Hedayaturahman Habibzai, and sayed tariq Pachakhan. Understanding of Valoctocogene Roxaparvovec (Roctavian) as a Breakthrough Gene Therapy for Hemophilia Type A. International Journal Of Advance Research And Innovative Ideas In Education [Internet]. 2023 [Cited : 06 December 2024]; 9(3) : 2150-2157. Available from: https://ijariie.com/AdminUploadPdf/Understanding_of_Valoctocogene_Roxaparvovec__Roctavian__as_a_Breakthrough_Gene_Therapy_for_Hemophilia_Type_A_ijariie20344.pdf
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