Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Results by year

Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1953 1
1964 1
1972 1
1975 3
1976 3
1977 4
1978 2
1979 5
1980 1
1981 5
1982 4
1983 7
1984 4
1985 12
1986 11
1987 10
1988 7
1989 8
1990 6
1991 12
1992 12
1993 15
1994 14
1995 10
1996 11
1997 13
1998 11
1999 15
2000 15
2001 15
2002 15
2003 21
2004 19
2005 16
2006 27
2007 19
2008 34
2009 36
2010 32
2011 47
2012 53
2013 45
2014 56
2015 40
2016 42
2017 41
2018 46
2019 38
2020 35
2021 41
2022 31
2023 27
2024 17

Text availability

Article attribute

Article type

Publication date

Search Results

882 results

Results by year

Filters applied: . Clear all
Quoted phrase not found in phrase index: "Hereditary factor IX deficiency disease"
Page 1
Gene therapy for hemophilia.
Nathwani AC. Nathwani AC. Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):569-578. doi: 10.1182/hematology.2022000388. Hematology Am Soc Hematol Educ Program. 2022. PMID: 36485127 Free PMC article. Review.
The cloning of the factor VIII (FVIII) and factor IX (FIX) genes in the 1980s has led to a succession of clinical advances starting with the advent of molecular diagnostic for hemophilia, followed by the development of recombinant clotting factor repla …
The cloning of the factor VIII (FVIII) and factor IX (FIX) genes in the 1980s has led to a succession of clinical advan …
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
Pipe SW, Leebeek FWG, Recht M, Key NS, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O'Connell N, Pasi KJ, Hart DP, Kazmi R, Astermark J, Hermans CRJR, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary SE, Escobar M, Gomez E, Kruse-Jarres R, Quon DV, Symington E, Wang M, Wheeler AP, Gut R, Liu YP, Dolmetsch RE, Cooper DL, Li Y, Goldstein B, Monahan PE. Pipe SW, et al. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644. N Engl J Med. 2023. PMID: 36812434 Clinical Trial.
BACKGROUND: Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby pr …
BACKGROUND: Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replaceme …
A diagnostic approach to mild bleeding disorders.
Boender J, Kruip MJ, Leebeek FW. Boender J, et al. J Thromb Haemost. 2016 Aug;14(8):1507-16. doi: 10.1111/jth.13368. Epub 2016 Jun 27. J Thromb Haemost. 2016. PMID: 27208505 Free article. Review.
Hemorrhagic diathesis can be caused by disorders in primary hemostasis (von Willebrand disease, inherited platelet function disorders), secondary hemostasis (hemophilia A and B, other (rare) coagulant factor deficiencies) and fibrinolysis, and in connective tissue o …
Hemorrhagic diathesis can be caused by disorders in primary hemostasis (von Willebrand disease, inherited platelet function disorders …
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 …
BACKGROUND: In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated …
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.
BACKGROUND: FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B. METHODS: In this mul …
BACKGROUND: FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid …
Inhibitors in haemophilia A and B: Management of bleeds, inhibitor eradication and strategies for difficult-to-treat patients.
Ljung R, Auerswald G, Benson G, Dolan G, Duffy A, Hermans C, Jiménez-Yuste V, Lambert T, Morfini M, Zupančić-Šalek S, Santagostino E. Ljung R, et al. Eur J Haematol. 2019 Feb;102(2):111-122. doi: 10.1111/ejh.13193. Epub 2018 Dec 6. Eur J Haematol. 2019. PMID: 30411401 Free PMC article. Review.
The standard therapy for patients with haemophilia is prophylactic treatment with replacement factor VIII (FVIII) or factor IX (FIX). Patients who develop inhibitors against FVIII/FIX face an increased risk of bleeding, and the likelihood of early development …
The standard therapy for patients with haemophilia is prophylactic treatment with replacement factor VIII (FVIII) or factor …
Recombinant factor IX.
White GC 2nd, Beebe A, Nielsen B. White GC 2nd, et al. Thromb Haemost. 1997 Jul;78(1):261-5. Thromb Haemost. 1997. PMID: 9198163 Review.
Despite the introduction of recombinant preparations of factor VIII and recombinant factor VII and VIIa, patients with other forms of hemophilia, especially hemophilia B, have remained at increased risk for blood borne viruses because of a lack of clin …
Despite the introduction of recombinant preparations of factor VIII and recombinant factor VII and VIIa, patients with other f …
Definitions in hemophilia: communication from the SSC of the ISTH.
Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Blanchette VS, et al. J Thromb Haemost. 2014 Nov;12(11):1935-9. doi: 10.1111/jth.12672. Epub 2014 Sep 3. J Thromb Haemost. 2014. PMID: 25059285 Free article. No abstract available.
Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results.
Shapiro AD, Angchaisuksiri P, Astermark J, Benson G, Castaman G, Chowdary P, Eichler H, Jiménez-Yuste V, Kavakli K, Matsushita T, Poulsen LH, Wheeler AP, Young G, Zupancic-Salek S, Oldenburg J. Shapiro AD, et al. Blood. 2019 Nov 28;134(22):1973-1982. doi: 10.1182/blood.2019001542. Blood. 2019. PMID: 31444162 Free PMC article. Clinical Trial.
PK/PD results were as expected, with no difference between hemophilia subtypes for concizumab exposure, free tissue factor pathway inhibitor, thrombin generation, prothrombin fragment 1+2, and d-dimers. ...Three patients had (very low to medium titer) ADA+ tests in each tr …
PK/PD results were as expected, with no difference between hemophilia subtypes for concizumab exposure, free tissue factor pathway in …
A Molecular Revolution in the Treatment of Hemophilia.
Butterfield JSS, Hege KM, Herzog RW, Kaczmarek R. Butterfield JSS, et al. Mol Ther. 2020 Apr 8;28(4):997-1015. doi: 10.1016/j.ymthe.2019.11.006. Epub 2019 Nov 13. Mol Ther. 2020. PMID: 31843450 Free PMC article. Review.
For decades, the monogenetic bleeding disorders hemophilia A and B (coagulation factor VIII and IX deficiency) have been treated with systemic protein replacement therapy. ...Adeno-associated virus (AAV) gene therapy provides lasting clotting factor re …
For decades, the monogenetic bleeding disorders hemophilia A and B (coagulation factor VIII and IX deficiency) have bee …
882 results