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19 results

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Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohort.
Pasi KJ, Lissitchkov T, Mamonov V, Mant T, Timofeeva M, Bagot C, Chowdary P, Georgiev P, Gercheva-Kyuchukova L, Madigan K, Van Nguyen H, Yu Q, Mei B, Benson CC, Ragni MV. Pasi KJ, et al. Among authors: benson cc. J Thromb Haemost. 2021 Jun;19(6):1436-1446. doi: 10.1111/jth.15270. Epub 2021 May 18. J Thromb Haemost. 2021. PMID: 33587824 Free PMC article. Clinical Trial.
BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.
Konkle BA, Shapiro AD, Quon DV, Staber JM, Kulkarni R, Ragni MV, Chhabra ES, Poloskey S, Rice K, Katragadda S, Fruebis J, Benson CC. Konkle BA, et al. Among authors: benson cc. N Engl J Med. 2020 Sep 10;383(11):1018-1027. doi: 10.1056/NEJMoa2002699. N Engl J Med. 2020. PMID: 32905674 Clinical Trial.
Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.
von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, Matino D, Klamroth R, Weyand AC, Jimenez-Yuste V, Nogami K, Poloskey S, Winding B, Willemze A, Knobe K; XTEND-1 Trial Group. von Drygalski A, et al. N Engl J Med. 2023 Jan 26;388(4):310-318. doi: 10.1056/NEJMoa2209226. N Engl J Med. 2023. PMID: 36720133 Clinical Trial.
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia.
Tran DQ, Benson CC, Boice JA, Chitlur M, Dunn AL, Escobar MA, Gupta K, Johnsen JM, Jorgenson J, Martin SD, Martin S, Meeks SL, Narvaez AA Jr, Quon DV, Reding MT, Reiss UM, Savage B, Schafer K, Steiner B, Thornburg C, Volland LM, von Drygalski A. Tran DQ, et al. Among authors: benson cc. Expert Rev Hematol. 2023 Mar;16(sup1):19-37. doi: 10.1080/17474086.2023.2171981. Expert Rev Hematol. 2023. PMID: 36920859 Free PMC article.
Mechanisms of Congenital Heart Disease Caused by NAA15 Haploinsufficiency.
Ward T, Tai W, Morton S, Impens F, Van Damme P, Van Haver D, Timmerman E, Venturini G, Zhang K, Jang MY, Willcox JAL, Haghighi A, Gelb BD, Chung WK, Goldmuntz E, Porter GA Jr, Lifton RP, Brueckner M, Yost HJ, Bruneau BG, Gorham J, Kim Y, Pereira A, Homsy J, Benson CC, DePalma SR, Varland S, Chen CS, Arnesen T, Gevaert K, Seidman C, Seidman JG. Ward T, et al. Among authors: benson cc. Circ Res. 2021 Apr 16;128(8):1156-1169. doi: 10.1161/CIRCRESAHA.120.316966. Epub 2021 Feb 9. Circ Res. 2021. PMID: 33557580 Free PMC article. Clinical Trial.
Small-Molecule Screen Identifies De Novo Nucleotide Synthesis as a Vulnerability of Cells Lacking SIRT3.
Gonzalez Herrera KN, Zaganjor E, Ishikawa Y, Spinelli JB, Yoon H, Lin JR, Satterstrom FK, Ringel A, Mulei S, Souza A, Gorham JM, Benson CC, Seidman JG, Sorger PK, Clish CB, Haigis MC. Gonzalez Herrera KN, et al. Among authors: benson cc. Cell Rep. 2018 Feb 20;22(8):1945-1955. doi: 10.1016/j.celrep.2018.01.076. Cell Rep. 2018. PMID: 29466723 Free PMC article.
HEART DISEASE. Titin mutations in iPS cells define sarcomere insufficiency as a cause of dilated cardiomyopathy.
Hinson JT, Chopra A, Nafissi N, Polacheck WJ, Benson CC, Swist S, Gorham J, Yang L, Schafer S, Sheng CC, Haghighi A, Homsy J, Hubner N, Church G, Cook SA, Linke WA, Chen CS, Seidman JG, Seidman CE. Hinson JT, et al. Among authors: benson cc. Science. 2015 Aug 28;349(6251):982-6. doi: 10.1126/science.aaa5458. Science. 2015. PMID: 26315439 Free PMC article.
19 results