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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.
Pharmacokinetics, Efficacy, and Safety of Nonacog Alfa in Previously Treated Patients with Moderately Severe to Severe Hemophilia B.
Korth-Bradley JM, Rendo P, Smith L, Altisent C. Korth-Bradley JM, et al. Clin Ther. 2016 Apr;38(4):936-44. doi: 10.1016/j.clinthera.2016.02.015. Epub 2016 Mar 8. Clin Ther. 2016. PMID: 26969334
IMPLICATIONS: The FIX activity profile after administration of nonacog alfa is predictable and is not altered after repeated exposure during usual hemophilia care. ...
IMPLICATIONS: The FIX activity profile after administration of nonacog alfa is predictable and is not altered after repeated exposure …
Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B.
Shah J, Kim H, Sivamurthy K, Monahan PE, Fries M. Shah J, et al. Curr Med Res Opin. 2023 Feb;39(2):227-237. doi: 10.1080/03007995.2022.2133492. Epub 2022 Oct 25. Curr Med Res Opin. 2023. PMID: 36285399
Bayesian model-based predictions of future participants suggest >80% would be free from prophylactic FIX replacement products 25.5 years post-infusion. Both models predicted FIX activity levels were not significantly influenced by pre-treatment AAV5 NAb status. . …
Bayesian model-based predictions of future participants suggest >80% would be free from prophylactic FIX replacement products 25.5 …