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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM. Nathwani AC, et al. N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309. N Engl J Med. 2014. PMID: 25409372 Free PMC article. Clinical Trial.
BACKGROUND: In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up to 16 months. ...RESULTS: A single intrave …
BACKGROUND: In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-ass …
Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.
Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Chowdary P, et al. N Engl J Med. 2022 Jul 21;387(3):237-247. doi: 10.1056/NEJMoa2119913. N Engl J Med. 2022. PMID: 35857660 Free article. Clinical Trial.
METHODS: In this multicenter, open-label, phase 1-2 trial, we assessed the safety and efficacy of varying doses of FLT180a in patients with severe or moderately severe hemophilia B (factor IX level, 2% of normal value). All the patients received glucocorticoi …
METHODS: In this multicenter, open-label, phase 1-2 trial, we assessed the safety and efficacy of varying doses of FLT180a in patients with …
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM. Nathwani AC, et al. N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10. N Engl J Med. 2011. PMID: 22149959 Free PMC article. Clinical Trial.
METHODS: We infused a single dose of a serotype-8-pseudotyped, self-complementary adenovirus-associated virus (AAV) vector expressing a codon-optimized human factor IX (FIX) transgene (scAAV2/8-LP1-hFIXco) in a peripheral vein in six patients with severe hemophilia …
METHODS: We infused a single dose of a serotype-8-pseudotyped, self-complementary adenovirus-associated virus (AAV) vector expressing a codo …
Etranacogene dezaparvovec for the treatment of adult patients with severe and moderately severe hemophilia B.
Castaman G, Coppens M, Pipe SW. Castaman G, et al. Expert Rev Hematol. 2023 Jul-Dec;16(12):919-932. doi: 10.1080/17474086.2023.2276206. Epub 2023 Dec 18. Expert Rev Hematol. 2023. PMID: 37882214 Review.
INTRODUCTION: Etranacogene dezaparvovec is the first gene therapy approved for treatment of adults with severe and moderately severe hemophilia B. AREAS COVERED: This review describes the results of the clinical trial program of AMT-060 and etranacogene dezap …
INTRODUCTION: Etranacogene dezaparvovec is the first gene therapy approved for treatment of adults with severe and moderately severe …
Molecular analysis of severe hemophilia B in Indian families: Identification of mutational hotspot and novel variants.
Agrawal N, Kumar R, Masih S, Srivastava P, Singh P, Jaiswal SK, Moirangthem A, Saxena D, Phadke SR, Mandal K. Agrawal N, et al. Int J Lab Hematol. 2022 Feb;44(1):186-192. doi: 10.1111/ijlh.13715. Epub 2021 Sep 29. Int J Lab Hematol. 2022. PMID: 34590426
METHOD: DNA was extracted from peripheral blood samples of 35 diagnosed severe hemophilia B patients belonging to 32 families, and were subjected to Sanger sequencing. ...CONCLUSIONS: This study provides a comprehensive mutational spectrum and mutation screen …
METHOD: DNA was extracted from peripheral blood samples of 35 diagnosed severe hemophilia B patients belonging to 32 fa …
Genetic Risk Factors and Inhibitor Development in Hemophilia: What Is Known and Searching for the Unknown.
Margaglione M, Intrieri M. Margaglione M, et al. Semin Thromb Hemost. 2018 Sep;44(6):509-516. doi: 10.1055/s-0038-1660816. Epub 2018 Jun 25. Semin Thromb Hemost. 2018. PMID: 29940657 Review.
In addition, modulation of the immunological response was acknowledged to play a pivotal role in the occurrence of inhibitors. However, with the exception of mutation testing in severe hemophilia B patients, no single risk factor or clinical score is currentl …
In addition, modulation of the immunological response was acknowledged to play a pivotal role in the occurrence of inhibitors. However, with …
Progress, and prospects in the therapeutic armamentarium of persons with congenital hemophilia. Defining the place for liver-directed gene therapy.
Di Minno G, Castaman G, De Cristofaro R, Brunetti-Pierri N, Pastore L, Castaldo G, Trama U, Di Minno M. Di Minno G, et al. Blood Rev. 2023 Mar;58:101011. doi: 10.1016/j.blre.2022.101011. Epub 2022 Aug 23. Blood Rev. 2023. PMID: 36031462 Review.
Once weekly/twice monthly infusions of EHL Factor IX (FIX) products achieve the same target in severe hemophilia B (HB). Gene therapy, which is likely to be licensed for clinical use within 1-2 years, embodies a shift beyond these standards. ...
Once weekly/twice monthly infusions of EHL Factor IX (FIX) products achieve the same target in severe hemophilia B (HB) …
Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B.
Shapiro AD, Kulkarni R, Ragni MV, Chambost H, Mahlangu J, Oldenburg J, Nolan B, Ozelo MC, Foster MC, Willemze A, Barnowski C, Jain N, Winding B, Dumont J, Lethagen S, Barnes C, Pasi KJ. Shapiro AD, et al. Blood Adv. 2023 Jul 11;7(13):3049-3057. doi: 10.1182/bloodadvances.2022009230. Blood Adv. 2023. PMID: 36848635 Free PMC article.
Long-term efficacy and safety of the extended half-life recombinant factor IX Fc fusion protein (rFIXFc) has been established among previously treated patients with severe hemophilia B in 2 phase 3 trials (B-LONG [#NCT01027364] and Kids B-LONG [#NCT01440946]) …
Long-term efficacy and safety of the extended half-life recombinant factor IX Fc fusion protein (rFIXFc) has been established among previous …
Costs and utilization of treatment in patients with hemophilia.
Rocha P, Carvalho M, Lopes M, Araújo F. Rocha P, et al. BMC Health Serv Res. 2015 Oct 26;15:484. doi: 10.1186/s12913-015-1134-3. BMC Health Serv Res. 2015. PMID: 26502954 Free PMC article.
The total mean aggregate cost per year (including clotting factor and hospital utilization) for patients with severe hemophilia B was 112,469, compared with 793 for mild hemophilia A. ...
The total mean aggregate cost per year (including clotting factor and hospital utilization) for patients with severe hemophilia …
Reduced bone density in individuals with severe hemophilia B.
Mansouritorghabeh H, Rezaieyazdi Z, Saadati N, Saghafi M, Mirfeizi Z, Rezai J. Mansouritorghabeh H, et al. Int J Rheum Dis. 2009 Jul;12(2):125-9. doi: 10.1111/j.1756-185X.2009.01394.x. Int J Rheum Dis. 2009. PMID: 20374329
In the current case-control study we tried to address bone mineral density in individuals with severe hemophilia B (decreased coagulation factor IX). METHODS: In our case-control study, we recruited bone density and biochemical indexes in 14 individuals with …
In the current case-control study we tried to address bone mineral density in individuals with severe hemophilia B (dec …
48 results