-
1
-
-
0021435842
-
The gene structure of human anti-haemophilic factor IX
-
Anson DS, Choo KH, Rees DJ, Giannelli F, Gould K, Huddleston JA, et al. The gene structure of human anti-haemophilic factor IX. EMBO Journal 1984;3:1053-60.
-
(1984)
EMBO Journal
, vol.3
, pp. 1053-1060
-
-
Anson, D.S.1
Choo, K.H.2
Rees, D.J.3
Giannelli, F.4
Gould, K.5
Huddleston, J.A.6
-
2
-
-
0027255865
-
HIV infection in haemophilia--a European cohort
-
[PUBMED: 8099271]
-
Aronstam A, Congard B, Evans DI, Gazengel CF, Herberg U, Hill FG, et al. HIV infection in haemophilia--a European cohort. Archives of Disease in Childhood 1993;68(4):521-4. [PUBMED: 8099271]
-
(1993)
Archives of Disease in Childhood
, vol.68
, Issue.4
, pp. 521-524
-
-
Aronstam, A.1
Congard, B.2
Evans, D.I.3
Gazengel, C.F.4
Herberg, U.5
Hill, F.G.6
-
3
-
-
0029011723
-
Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A
-
Bi L, Lawler AM, Antonarakis SE, High KA, Gearhart JD, Kazazian HH Jr. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nature Genetics 1995;10(1):119-21.
-
(1995)
Nature Genetics
, vol.10
, Issue.1
, pp. 119-121
-
-
Bi, L.1
Lawler, A.M.2
Antonarakis, S.E.3
High, K.A.4
Gearhart, J.D.5
Kazazian, H.H.6
-
4
-
-
0014431093
-
A new high-potency glycine-precipitated antihemophilic factor (AHF) concentrate. Treatment of classical hemophilia and hemophilia with inhibitors
-
Brinkhous KM, Shanbrom E, Roberts HR, Webster WP, Fekete L, Wagner RH. A new high-potency glycine-precipitated antihemophilic factor (AHF) concentrate. Treatment of classical hemophilia and hemophilia with inhibitors. JAMA 1968;205(9):613-7.
-
(1968)
JAMA
, vol.205
, Issue.9
, pp. 613-617
-
-
Brinkhous, K.M.1
Shanbrom, E.2
Roberts, H.R.3
Webster, W.P.4
Fekete, L.5
Wagner, R.H.6
-
5
-
-
84899562558
-
Changing paradigm of prophylaxis with longer acting factor concentrates
-
[PUBMED: 24762284]
-
Carcao M. Changing paradigm of prophylaxis with longer acting factor concentrates. Haemophilia 2014;20 Suppl 4:99-105. [PUBMED: 24762284]
-
(2014)
Haemophilia
, vol.20
, pp. 99-105
-
-
Carcao, M.1
-
6
-
-
37449021072
-
Desmopressin for the treatment of haemophilia
-
Castaman G. Desmopressin for the treatment of haemophilia. Haemophilia 2008;14 Suppl 1:15-20.
-
(2008)
Haemophilia
, vol.14
, pp. 15-20
-
-
Castaman, G.1
-
7
-
-
0019957533
-
Molecular cloning of the gene for human anti-haemophilic factor IX
-
Choo KH, Gould KG, Rees DJ, Brownlee GG. Molecular cloning of the gene for human anti-haemophilic factor IX. Nature 1982;299(5879):178-80.
-
(1982)
Nature
, vol.299
, Issue.5879
, pp. 178-180
-
-
Choo, K.H.1
Gould, K.G.2
Rees, D.J.3
Brownlee, G.G.4
-
8
-
-
33750049639
-
The tragic history of AIDS in the hemophilia population, 1982-1984
-
Evatt BL. The tragic history of AIDS in the hemophilia population, 1982-1984. Journal of Thrombosis and Haemostasis 2006;4(11):2295-301.
-
(2006)
Journal of Thrombosis and Haemostasis
, vol.4
, Issue.11
, pp. 2295-2301
-
-
Evatt, B.L.1
-
9
-
-
85006785146
-
Preliminary results of a phase 1/2 trial of SPK-9001, a hyperactive FIX variant delivered by a novel capsid, demonstrate consistent factor IX activity levels at the lowest dose cohort
-
George LA, Sullivan S, Teitel J, Cuker A, Luk A, Wright F, et al. Preliminary results of a phase 1/2 trial of SPK-9001, a hyperactive FIX variant delivered by a novel capsid, demonstrate consistent factor IX activity levels at the lowest dose cohort. Haemophilia 2016;22(Suppl 4):151-2. [DOI: 10.1111/hae.13068]
-
(2016)
Haemophilia
, vol.22
, pp. 151-152
-
-
George, L.A.1
Sullivan, S.2
Teitel, J.3
Cuker, A.4
Luk, A.5
Wright, F.6
-
10
-
-
0020526898
-
Generalized lymphadenopathy and T cell abnormalities in hemophilia A
-
[PUBMED: 6223133]
-
Gill JC, Menitove JE, Wheeler D, Aster RH, Montgomery RR. Generalized lymphadenopathy and T cell abnormalities in hemophilia A. Journal of Pediatrics 1983;103(1):18-22. [PUBMED: 6223133]
-
(1983)
Journal of Pediatrics
, vol.103
, Issue.1
, pp. 18-22
-
-
Gill, J.C.1
Menitove, J.E.2
Wheeler, D.3
Aster, R.H.4
Montgomery, R.R.5
-
11
-
-
0021750055
-
Characterization of the human factor VIII gene
-
Gitschier J, Wood WI, Goralka TM, Wion KL, Chen EY, Eaton DH, et al. Characterization of the human factor VIII gene. Nature 1984;312:326-30.
-
(1984)
Nature
, vol.312
, pp. 326-330
-
-
Gitschier, J.1
Wood, W.I.2
Goralka, T.M.3
Wion, K.L.4
Chen, E.Y.5
Eaton, D.H.6
-
12
-
-
0024317522
-
A prospective study of human immunodeficiency virus type 1 infection and the development of AIDS in subjects with hemophilia
-
[PUBMED: 2477702]
-
Goedert JJ, Kessler CM, Aledort LM, Biggar RJ, Andes WA, White GC 2nd, et al. A prospective study of human immunodeficiency virus type 1 infection and the development of AIDS in subjects with hemophilia. New England Journal of Medicine 1989;321(17):1141-8. [PUBMED: 2477702]
-
(1989)
New England Journal of Medicine
, vol.321
, Issue.17
, pp. 1141-1148
-
-
Goedert, J.J.1
Kessler, C.M.2
Aledort, L.M.3
Biggar, R.J.4
Andes, W.A.5
White, G.C.6
-
13
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
-
Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302(5644):415-9.
-
(2003)
Science
, vol.302
, Issue.5644
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
McCormack, M.P.4
Wulffraat, N.5
Leboulch, P.6
-
14
-
-
70049099036
-
Chapter 8: Assessing risk of bias in included studies. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011
-
Higgins JPT, Altman DG, Sterne JAC, on behalf of the Cochrane Statistical Methods Group and the Cochrane Bias Methods Group. Chapter 8: Assessing risk of bias in included studies. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
-
-
-
Higgins, J.P.T.1
Altman, D.G.2
Sterne, J.A.C.3
-
15
-
-
84966694802
-
Chapter 16: Special topics in statistics. In: Higgins JPT, Green S (editors). Cochrane Handbook of Systematic Reviews of Interventions. Version 5.1 [updated March 2011]. The Cochrane Collaboration, 2011
-
Higgins JPT, Deeks JJ, Altman DG. Chapter 16: Special topics in statistics. In: Higgins JPT, Green S (editors). Cochrane Handbook of Systematic Reviews of Interventions. Version 5.1 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
-
-
-
Higgins, J.P.T.1
Deeks, J.J.2
Altman, D.G.3
-
16
-
-
79960649903
-
Gene therapy for haemophilia: a long and winding road
-
High KA. Gene therapy for haemophilia: a long and winding road. Journal of Thrombosis and Haemostasis 2011;9 Suppl 1:2-11.
-
(2011)
Journal of Thrombosis and Haemostasis
, vol.9
, pp. 2-11
-
-
High, K.A.1
-
17
-
-
80055081959
-
Clotting factor concentrates given to prevent bleeding and bleeding-related complications in people with hemophilia A or B
-
[PUBMED: 21901684]
-
Iorio A, Marchesini E, Marcucci M, Stobart K, Chan AK. Clotting factor concentrates given to prevent bleeding and bleeding-related complications in people with hemophilia A or B. Cochrane Database of Systematic Reviews 2011, Issue 9. [DOI: 10.1002/14651858.CD003429.pub4; PUBMED: 21901684]
-
(2011)
Cochrane Database of Systematic Reviews
, Issue.9
-
-
Iorio, A.1
Marchesini, E.2
Marcucci, M.3
Stobart, K.4
Chan, A.K.5
-
18
-
-
84912400091
-
Hepatitis and clotting-factor concentrates
-
[PUBMED: 5067966]
-
Kasper CK, Kipnis SA. Hepatitis and clotting-factor concentrates. JAMA 1972;221(5):510. [PUBMED: 5067966]
-
(1972)
JAMA
, vol.221
, Issue.5
, pp. 510
-
-
Kasper, C.K.1
Kipnis, S.A.2
-
19
-
-
84942546645
-
Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A
-
[PUBMED: 26157075]
-
Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, Mant T, Shima M, et al. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood 2015;126(9):1078-85. [DOI: 10.1182/blood-2015-03-630897; PUBMED: 26157075]
-
(2015)
Blood
, vol.126
, Issue.9
, pp. 1078-1085
-
-
Konkle, B.A.1
Stasyshyn, O.2
Chowdary, P.3
Bevan, D.H.4
Mant, T.5
Shima, M.6
-
20
-
-
0035023768
-
High-dose DDAVP intranasal spray (Stimate) for the prevention and treatment of bleeding in patients with mild haemophilia A, mild or moderate type 1 von Willebrand disease and symptomatic carriers of haemophilia A
-
Leissinger C, Becton D, Cornell C Jr, Cox Gill J. High-dose DDAVP intranasal spray (Stimate) for the prevention and treatment of bleeding in patients with mild haemophilia A, mild or moderate type 1 von Willebrand disease and symptomatic carriers of haemophilia A. Haemophilia 2001;7(3):258-66.
-
(2001)
Haemophilia
, vol.7
, Issue.3
, pp. 258-266
-
-
Leissinger, C.1
Becton, D.2
Cornell, C.3
Cox Gill, J.4
-
21
-
-
0025268925
-
Hepatitis C antibody and chronic liver disease in haemophilia
-
[PUBMED: 1971863]
-
Makris M, Preston FE, Triger DR, Underwood JC, Choo QL, Kuo G, et al. Hepatitis C antibody and chronic liver disease in haemophilia. Lancet 1990;335(8698):1117-9. [PUBMED: 1971863]
-
(1990)
Lancet
, vol.335
, Issue.8698
, pp. 1117-1119
-
-
Makris, M.1
Preston, F.E.2
Triger, D.R.3
Underwood, J.C.4
Choo, Q.L.5
Kuo, G.6
-
22
-
-
34547757915
-
Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia
-
Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. New England Journal of Medicine 2007;357(6):535-44.
-
(2007)
New England Journal of Medicine
, vol.357
, Issue.6
, pp. 535-544
-
-
Manco-Johnson, M.J.1
Abshire, T.C.2
Shapiro, A.D.3
Riske, B.4
Hacker, M.R.5
Kilcoyne, R.6
-
23
-
-
0013442744
-
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
-
Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 2003; Vol. 101, issue 8:2963-72.
-
(2003)
Blood
, vol.101
, Issue.8
, pp. 2963-2972
-
-
Manno, C.S.1
Chew, A.J.2
Hutchison, S.3
Larson, P.J.4
Herzog, R.W.5
Arruda, V.R.6
-
24
-
-
33644820684
-
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
-
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nature Medicine 2006;12(3):342-7.
-
(2006)
Nature Medicine
, vol.12
, Issue.3
, pp. 342-347
-
-
Manno, C.S.1
Pierce, G.F.2
Arruda, V.R.3
Glader, B.4
Ragni, M.5
Rasko, J.J.6
-
25
-
-
0017336844
-
1-Deamino-8-d-arginine vasopressin: a new pharmacological approach to the management of haemophilia and von Willebrands' diseases
-
[PUBMED: 67283]
-
Mannucci PM, Ruggeri ZM, Pareti FI, Capitanio A. 1-Deamino-8-d-arginine vasopressin: a new pharmacological approach to the management of haemophilia and von Willebrands' diseases. Lancet 1977;1(8017):869-72. [PUBMED: 67283]
-
(1977)
Lancet
, vol.1
, Issue.8017
, pp. 869-872
-
-
Mannucci, P.M.1
Ruggeri, Z.M.2
Pareti, F.I.3
Capitanio, A.4
-
26
-
-
85006791028
-
Updated results from a dose escalating study in adult patients with haemophilia B treated with AMT-060 (AAV5-hFIX) gene therapy
-
Miesbach W, Tangelder M, Klamroth R, Schutgens R, Coppens M, Kampmann P, et al. Updated results from a dose escalating study in adult patients with haemophilia B treated with AMT-060 (AAV5-hFIX) gene therapy. Haemophilia 2016;22(Suppl 4):151-2. [DOI: 10.1111/hae.13068]
-
(2016)
Haemophilia
, vol.22
, pp. 151-152
-
-
Miesbach, W.1
Tangelder, M.2
Klamroth, R.3
Schutgens, R.4
Coppens, M.5
Kampmann, P.6
-
27
-
-
23844556421
-
Large-scale analysis of adeno-associated virus vector integration sites in normal human cells
-
Miller DG, Trobridge GD, Petek LM, Jacobs MA, Kaul R, Russell DW. Large-scale analysis of adeno-associated virus vector integration sites in normal human cells. Journal of Virology 2005;79(17):11434-42.
-
(2005)
Journal of Virology
, vol.79
, Issue.17
, pp. 11434-11442
-
-
Miller, D.G.1
Trobridge, G.D.2
Petek, L.M.3
Jacobs, M.A.4
Kaul, R.5
Russell, D.W.6
-
28
-
-
79954622209
-
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
-
Mingozzi F, High KA. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nature Reviews. Genetics 2011;12(5):341-55.
-
(2011)
Nature Reviews. Genetics
, vol.12
, Issue.5
, pp. 341-355
-
-
Mingozzi, F.1
High, K.A.2
-
29
-
-
14744268153
-
Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver
-
Nakai H, Wu X, Fuess S, Storm TA, Munroe D, Montini E, et al. Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver. Journal of Virology 2005;79(6):3606-14.
-
(2005)
Journal of Virology
, vol.79
, Issue.6
, pp. 3606-3614
-
-
Nakai, H.1
Wu, X.2
Fuess, S.3
Storm, T.A.4
Munroe, D.5
Montini, E.6
-
31
-
-
33645528206
-
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
-
Nathwani AC, Gray JT, Ng CY, Zhou J, Spence Y, Waddington SN, et al. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 2006;107(7):2653-61.
-
(2006)
Blood
, vol.107
, Issue.7
, pp. 2653-2661
-
-
Nathwani, A.C.1
Gray, J.T.2
Ng, C.Y.3
Zhou, J.4
Spence, Y.5
Waddington, S.N.6
-
32
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
-
[PUBMED: 22149959]
-
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. New England Journal of Medicine 2011;365(25):2357-65. [PUBMED: 22149959]
-
(2011)
New England Journal of Medicine
, vol.365
, Issue.25
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.2
Rangarajan, S.3
Rosales, C.4
McIntosh, J.5
Linch, D.C.6
-
33
-
-
84911383748
-
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
-
[PUBMED: 25409372]
-
Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. New England Journal of Medicine 2014;371(21):1994-2004. [DOI: 10.1056/NEJMoa1407309; PUBMED: 25409372]
-
(2014)
New England Journal of Medicine
, vol.371
, Issue.21
, pp. 1994-2004
-
-
Nathwani, A.C.1
Reiss, U.M.2
Tuddenham, E.G.3
Rosales, C.4
Chowdary, P.5
McIntosh, J.6
-
34
-
-
84875561330
-
Study of ataluren (PTC124™) in cystic fibrosis
-
(accessed 1 September 2014). [NCT00803205]
-
NCT00803205 Study of ataluren (PTC124™) in cystic fibrosis. http://clinicaltrials.gov/ct2/show/study/NCT00803205 (accessed 1 September 2014). [NCT00803205]
-
-
-
-
35
-
-
85006772714
-
Phase 2b extension study of ataluren (PTC124) in Duchenne/Becker muscular dystrophy (DMD/BMD)
-
(accessed 1 September 2014). [NCT00847379]
-
NCT00847379 Phase 2b extension study of ataluren (PTC124) in Duchenne/Becker muscular dystrophy (DMD/BMD). http://clinicaltrials.gov/ct2/show/NCT00847379 (accessed 1 September 2014). [NCT00847379]
-
-
-
-
36
-
-
85041513211
-
Study of ataluren (PTC124®) in hemophilia A and B
-
(accessed 1 September 2014). [NCT00947193]
-
NCT00947193 Study of ataluren (PTC124®) in hemophilia A and B. http://clinicaltrials.gov/ct2/show/NCT00947193 (accessed 1 September 2014). [NCT00947193]
-
-
-
-
37
-
-
85006822256
-
An open label dose-escalation study of a self complementary adeno-associated viral vector (scAAV 2/8-LP1-hFIXco) for gene transfer in hemophilia B
-
(accessed 26 April 2014). [NCT00979238]
-
NCT00979238 An open label dose-escalation study of a self complementary adeno-associated viral vector (scAAV 2/8-LP1-hFIXco) for gene transfer in hemophilia B. http://clinicaltrials.gov/show/NCT00979238 (accessed 26 April 2014). [NCT00979238]
-
-
-
-
38
-
-
85006829006
-
A Phase 1 safety study in subjects with severe hemophilia B (factor IX deficiency) using a single-stranded, adeno-associated pseudotype 8 viral vector to deliver the gene for human factor IX
-
(accessed 26 April 2014). [NCT01620801]
-
NCT01620801 A Phase 1 safety study in subjects with severe hemophilia B (factor IX deficiency) using a single-stranded, adeno-associated pseudotype 8 viral vector to deliver the gene for human factor IX. http://clinicaltrials.gov/show/NCT01620801 (accessed 26 April 2014). [NCT01620801]
-
-
-
-
39
-
-
85006785170
-
A phase 1/2 open-label, single ascending dose trial of a self-complementing optimized adeno-associated virus serotype 8 factor IX gene therapy (AskBio009) in adults with hemophilia B
-
(accessed 26 April 2014). [NCT01687608]
-
NCT01687608 A phase 1/2 open-label, single ascending dose trial of a self-complementing optimized adeno-associated virus serotype 8 factor IX gene therapy (AskBio009) in adults with hemophilia B. http://clinicaltrials.gov/show/NCT01687608 (accessed 26 April 2014). [NCT01687608]
-
-
-
-
40
-
-
85006828994
-
Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
-
(accessed 05 November 2016). [NCT02396342]
-
NCT02396342 Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B. https://clinicaltrials.gov/ct2/show/NCT02396342 (accessed 05 November 2016). [NCT02396342]
-
-
-
-
41
-
-
85006812455
-
A Gene Therapy Study for Hemophilia B
-
(accessed 05 November 2016). [NCT02484092]
-
NCT02484092 A Gene Therapy Study for Hemophilia B. https://clinicaltrials.gov/ct2/show/NCT02484092 (accessed 05 November 2016). [NCT02484092]
-
-
-
-
42
-
-
85006716132
-
Gene Therapy Study in Severe Haemophilia A Patients
-
(accessed 05 November 2016). [NCT02576795]
-
NCT02576795 Gene Therapy Study in Severe Haemophilia A Patients. https://clinicaltrials.gov/ct2/show/NCT02576795 (accessed 05 November 2016). [NCT02576795]
-
-
-
-
43
-
-
85006816046
-
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
-
(accessed 05 November 2016). [NCT02618915]
-
NCT02618915 Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B. https://clinicaltrials.gov/ct2/show/NCT02618915 (accessed 05 November 2016). [NCT02618915]
-
-
-
-
44
-
-
85006816065
-
Ascending Dose Study of Genome Editing by the Zinc Finger Protein (ZFP) Therapeutic SB-FIX in Severe Hemophilia B
-
(accessed 05 November 2016). [NCT02695160]
-
NCT02695160 Ascending Dose Study of Genome Editing by the Zinc Finger Protein (ZFP) Therapeutic SB-FIX in Severe Hemophilia B. https://clinicaltrials.gov/ct2/show/NCT02695160 (accessed 05 November 2016). [NCT02695160]
-
-
-
-
45
-
-
52049089405
-
Fast Facts
-
(accessed 26 April 2014)
-
National Hemophilia Foundation. Fast Facts. http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=259&contentid=476 (accessed 26 April 2014).
-
-
-
-
46
-
-
0024565121
-
Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B
-
Palmer TD, Thompson AR, Miller AD. Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B. Blood 1989;73(2):438-45.
-
(1989)
Blood
, vol.73
, Issue.2
, pp. 438-445
-
-
Palmer, T.D.1
Thompson, A.R.2
Miller, A.D.3
-
47
-
-
85011356650
-
Interim results of an open-label, phase 1/2 study of BMN 270, an AAV5- FVIII gene transfer in severe hemophilia A
-
Pas J, Wong W, Rangarajan S, Wilde J, Perry D, Madan B, et al. Interim results of an open-label, phase 1/2 study of BMN 270, an AAV5- FVIII gene transfer in severe hemophilia A. Haemophilia 2016;22(Suppl 4):151-2. [DOI: 10.1111/hae.13068]
-
(2016)
Haemophilia
, vol.22
, pp. 151-152
-
-
Pas, J.1
Wong, W.2
Rangarajan, S.3
Wilde, J.4
Perry, D.5
Madan, B.6
-
48
-
-
43149101868
-
Walk a mile in the moccasins of people with haemophilia
-
Ponder KP, Srivastava A. Walk a mile in the moccasins of people with haemophilia. Haemophilia 2008;14(3):618-20.
-
(2008)
Haemophilia
, vol.14
, Issue.3
, pp. 618-620
-
-
Ponder, K.P.1
Srivastava, A.2
-
49
-
-
84855166312
-
Merry christmas for patients with hemophilia B
-
Ponder KP. Merry christmas for patients with hemophilia B. New England Journal of Medicine 2011;365(25):2424-5.
-
(2011)
New England Journal of Medicine
, vol.365
, Issue.25
, pp. 2424-2425
-
-
Ponder, K.P.1
-
50
-
-
0141679053
-
Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion
-
Powell JS, Ragni MV, White GC 2nd, Lusher JM, Hillman-Wiseman C, Moon TE, et al. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood 2003;102(6):2038-45.
-
(2003)
Blood
, vol.102
, Issue.6
, pp. 2038-2045
-
-
Powell, J.S.1
Ragni, M.V.2
White, G.C.3
Lusher, J.M.4
Hillman-Wiseman, C.5
Moon, T.E.6
-
51
-
-
84889769562
-
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B
-
[PUBMED: 24304002]
-
Powell JS, Pasi KJ, Ragni MV, Ozelo MC, Valentino LA, Mahlangu JN, et al. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. New England Journal of Medicine 2013;369(24):2313-23. [DOI: 10.1056/NEJMoa1305074; PUBMED: 24304002]
-
(2013)
New England Journal of Medicine
, vol.369
, Issue.24
, pp. 2313-2323
-
-
Powell, J.S.1
Pasi, K.J.2
Ragni, M.V.3
Ozelo, M.C.4
Valentino, L.A.5
Mahlangu, J.N.6
-
52
-
-
0035822005
-
Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A
-
Roth DA, Tawa NE Jr, O'Brien JM, Treco DA, Selden RF, Factor VIII Transkaryotic Therapy Study Group. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. New England Journal of Medicine 2001;344(23):1735-42.
-
(2001)
New England Journal of Medicine
, vol.344
, Issue.23
, pp. 1735-1742
-
-
Roth, D.A.1
Tawa, N.E.2
O'Brien, J.M.3
Treco, D.A.4
Selden, R.F.5
-
53
-
-
73949125216
-
Venous access in haemophilic children: choice and management
-
[PUBMED: 20059565]
-
Santagostino E, Mancuso ME. Venous access in haemophilic children: choice and management. Haemophilia 2010;16 Suppl 1:20-4. [DOI: 10.1111/j.1365-2516.2009.02156.x; PUBMED: 20059565]
-
(2010)
Haemophilia
, vol.16
, pp. 20-24
-
-
Santagostino, E.1
Mancuso, M.E.2
-
54
-
-
84963542247
-
Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial
-
[PUBMED: 26755710]
-
Santagostino E, Martinowitz U, Lissitchkov T, Pan-Petesch B, Hanabusa H, Oldenburg J, et al. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood 2016;127(14):1761-9. [DOI: 10.1182/blood-2015-09-669234; PUBMED: 26755710]
-
(2016)
Blood
, vol.127
, Issue.14
, pp. 1761-1769
-
-
Santagostino, E.1
Martinowitz, U.2
Lissitchkov, T.3
Pan-Petesch, B.4
Hanabusa, H.5
Oldenburg, J.6
-
55
-
-
84890730197
-
Chapter 10: Addressing reporting biases. In: Higgins JPT, Green S (editors). Cochrane Handbook forSystematic Reviews of Interventions Version 5.1 [updated March 2011]. The Cochrane Collaboration, 2011
-
Sterne JAC, Egger M, Moher D, on behalf of the Cochrane Bias Methods Group. Chapter 10: Addressing reporting biases. In: Higgins JPT, Green S (editors). Cochrane Handbook forSystematic Reviews of Interventions Version 5.1 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
-
-
-
Sterne, J.A.C.1
Egger, M.2
Moher, D.3
-
56
-
-
0021715169
-
Molecular cloning of a cDNA encoding human antihaemophilic factor
-
Toole JJ, Knopf JL, Wozney JM, Sultzman LA, Buecker JL, Pittman DD, et al. Molecular cloning of a cDNA encoding human antihaemophilic factor. Nature 1984;312(5992):342-7.
-
(1984)
Nature
, vol.312
, Issue.5992
, pp. 342-347
-
-
Toole, J.J.1
Knopf, J.L.2
Wozney, J.M.3
Sultzman, L.A.4
Buecker, J.L.5
Pittman, D.D.6
-
57
-
-
84862882940
-
Gene therapy for haemophilia B
-
Tuddenham E. Gene therapy for haemophilia B. Haemophilia 2012;18 Suppl 4:13-7.
-
(2012)
Haemophilia
, vol.18
, pp. 13-17
-
-
Tuddenham, E.1
-
58
-
-
0033621126
-
Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice
-
VandenDriessche T, Vanslembrouck V, Goovaerts I, Zwinnen H, Vanderhaeghen ML, Collen D, et al. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice. Proceedings of the National Academy of Sciences of the United States of America 1999;96(18):10379-84.
-
(1999)
Proceedings of the National Academy of Sciences of the United States of America
, vol.96
, Issue.18
, pp. 10379-10384
-
-
VandenDriessche, T.1
Vanslembrouck, V.2
Goovaerts, I.3
Zwinnen, H.4
Vanderhaeghen, M.L.5
Collen, D.6
-
59
-
-
0013829369
-
Clinical use of a new glycine-precipitated antihemophilic fraction
-
Webster WP, Roberts HR, Thelin GM, Wagner RH, Brinkhous KM. Clinical use of a new glycine-precipitated antihemophilic fraction. American Journal of the Medical Sciences 1965;250(6):643-51.
-
(1965)
American Journal of the Medical Sciences
, vol.250
, Issue.6
, pp. 643-651
-
-
Webster, W.P.1
Roberts, H.R.2
Thelin, G.M.3
Wagner, R.H.4
Brinkhous, K.M.5
-
60
-
-
84902151512
-
Annual Report 2013
-
(accessed 02 December 2016)
-
World Federation of Hemophilia. Annual Report 2013. http://www1.wfh.org/publications/files/pdf-1578.pdf (accessed 02 December 2016).
-
-
-
-
61
-
-
79951616770
-
Current options and new developments in the treatment of haemophilia
-
Wong T, Recht M. Current options and new developments in the treatment of haemophilia. Drugs 2011;71(3):305-20.
-
(2011)
Drugs
, vol.71
, Issue.3
, pp. 305-320
-
-
Wong, T.1
Recht, M.2
-
62
-
-
0037589022
-
Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B
-
Xu L, Gao C, Sands MS, Cai SR, Nichols TC, Bellinger DA, et al. Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B. Blood 2003;101(10):3924-32.
-
(2003)
Blood
, vol.101
, Issue.10
, pp. 3924-3932
-
-
Xu, L.1
Gao, C.2
Sands, M.S.3
Cai, S.R.4
Nichols, T.C.5
Bellinger, D.A.6
-
63
-
-
84930179981
-
Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A
-
[PUBMED: 25912075]
-
Young G, Mahlangu J, Kulkarni R, Nolan B, Liesner R, Pasi J, et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A. Journal of Thrombosis and Haemostasis 2015;13(6):967-77. [DOI: 10.1111/jth.12911; PUBMED: 25912075]
-
(2015)
Journal of Thrombosis and Haemostasis
, vol.13
, Issue.6
, pp. 967-977
-
-
Young, G.1
Mahlangu, J.2
Kulkarni, R.3
Nolan, B.4
Liesner, R.5
Pasi, J.6
-
64
-
-
84922232199
-
Gene therapy for haemophilia
-
Sharma A, Easow Mathew M, Sriganesh V, Neely JA, Kalipatnapu S. Gene therapy for haemophilia. Cochrane Database of Systematic Reviews 2014, Issue 11. [DOI: 10.1002/14651858.CD010822.pub2]
-
(2014)
Cochrane Database of Systematic Reviews
, Issue.11
-
-
Sharma, A.1
Easow Mathew, M.2
Sriganesh, V.3
Neely, J.A.4
Kalipatnapu, S.5
|