-
1
-
-
0035822038
-
The hemophilias-from royal genes to gene therapy
-
Mannucci PM, Tuddenham EG. The hemophilias-from royal genes to gene therapy. N Engl J Med. 2001; 344:1773-1779.
-
(2001)
N Engl J Med.
, vol.344
, pp. 1773-1779
-
-
Mannucci, P.M.1
Tuddenham, E.G.2
-
2
-
-
47749085063
-
Inherited bleeding disorders: results from the Italian Regional Haemophilia Centre of Pescara
-
Dragani A, Malizia R, Iuliani O, Di M I, Davi G. Inherited bleeding disorders: results from the Italian Regional Haemophilia Centre of Pescara. Blood Transfus. 2008; 6:136-142.
-
(2008)
Blood Transfus.
, vol.6
, pp. 136-142
-
-
Dragani, A.1
Malizia, R.2
Iuliani, O.3
Di, M.I.4
Davi, G.5
-
4
-
-
20344405733
-
The spectrum of mutations in Southern Spanish patients with hemophilia A and identification of 28 novel mutations
-
Fernandez-Lopez O, Garcia-Lozano JR, Nunez-Vazquez R, Perez-Garrido R, Nunez-Roldan A. The spectrum of mutations in Southern Spanish patients with hemophilia A and identification of 28 novel mutations. Haematologica. 2005; 90:707-710.
-
(2005)
Haematologica.
, vol.90
, pp. 707-710
-
-
Fernandez-Lopez, O.1
Garcia-Lozano, J.R.2
Nunez-Vazquez, R.3
Perez-Garrido, R.4
Nunez-Roldan, A.5
-
5
-
-
33745749037
-
The spectrum of mutations and molecular pathogenesis of hemophilia A in 181 Portuguese patients
-
David D, Ventura C, Moreira I, et al. The spectrum of mutations and molecular pathogenesis of hemophilia A in 181 Portuguese patients. Haematologica. 2006; 91:840-843.
-
(2006)
Haematologica.
, vol.91
, pp. 840-843
-
-
David, D.1
Ventura, C.2
Moreira, I.3
-
6
-
-
51249105747
-
Haemophilia A mutations in the UK: results of screening one-third of the population
-
Green PM, Bagnall RD, Waseem NH, Giannelli F. Haemophilia A mutations in the UK: results of screening one-third of the population. Br J Haematol. 2008; 143:115-128.
-
(2008)
Br J Haematol.
, vol.143
, pp. 115-128
-
-
Green, P.M.1
Bagnall, R.D.2
Waseem, N.H.3
Giannelli, F.4
-
7
-
-
43449104265
-
The Italian AICE-Genetics hemophilia A database: results and correlation with clinical phenotype
-
Margaglione M, Castaman G, Morfini M, et al. The Italian AICE-Genetics hemophilia A database: results and correlation with clinical phenotype. Haematologica. 2008; 93:722-728.
-
(2008)
Haematologica.
, vol.93
, pp. 722-728
-
-
Margaglione, M.1
Castaman, G.2
Morfini, M.3
-
8
-
-
84880473815
-
Correlation between phenotype and genotype in a large unselected cohort of children with severe hemophilia A
-
S1.
-
Carcao MD, Van Den Berg HM, Ljung R, Mancuso ME. Correlation between phenotype and genotype in a large unselected cohort of children with severe hemophilia A. Blood. 2013; 121:3946-3952, S1.
-
(2013)
Blood.
, vol.121
, pp. 3946-3952
-
-
Carcao, M.D.1
Van Den Berg, H.M.2
Ljung, R.3
Mancuso, M.E.4
-
9
-
-
18344387131
-
Results of an orthopaedic survey in young patients with severe haemophilia in Spain
-
Tusell JM, Aznar JA, Querol F, Quintana M, Moreno M, Gorina E. Results of an orthopaedic survey in young patients with severe haemophilia in Spain. Haemophilia. 2002; 8(Suppl 2):38-42.
-
(2002)
Haemophilia.
, vol.8
, pp. 38-42
-
-
Tusell, J.M.1
Aznar, J.A.2
Querol, F.3
Quintana, M.4
Moreno, M.5
Gorina, E.6
-
10
-
-
12144290833
-
Joint range-of-motion limitations among young males with hemophilia: prevalence and risk factors
-
Soucie JM, Cianfrini C, Janco RL, et al. Joint range-of-motion limitations among young males with hemophilia: prevalence and risk factors. Blood. 2004; 103:2467-2473.
-
(2004)
Blood.
, vol.103
, pp. 2467-2473
-
-
Soucie, J.M.1
Cianfrini, C.2
Janco, R.L.3
-
11
-
-
33646003862
-
Pain and functional limitations in patients with severe haemophilia
-
van Genderen FR, Fischer K, Heijnen L, et al. Pain and functional limitations in patients with severe haemophilia. Haemophilia. 2006; 12:147-153.
-
(2006)
Haemophilia.
, vol.12
, pp. 147-153
-
-
van Genderen, F.R.1
Fischer, K.2
Heijnen, L.3
-
12
-
-
79951939019
-
Perceived competence in children and adolescents with haemophilia: an explorative study
-
Hegeman AK, van Genderen FR, Meijer S, Van Den Briel MM, Tamminga RY, Van WE. Perceived competence in children and adolescents with haemophilia: an explorative study. Haemophilia. 2011; 17:81-89.
-
(2011)
Haemophilia.
, vol.17
, pp. 81-89
-
-
Hegeman, A.K.1
van Genderen, F.R.2
Meijer, S.3
Van Den Briel, M.M.4
Tamminga, R.Y.5
Van, W.E.6
-
13
-
-
41349105729
-
Social participation of patients with hemophilia in the Netherlands
-
Plug I, Peters M, Mauser-Bunschoten EP, et al. Social participation of patients with hemophilia in the Netherlands. Blood. 2008; 111:1811-1815.
-
(2008)
Blood.
, vol.111
, pp. 1811-1815
-
-
Plug, I.1
Peters, M.2
Mauser-Bunschoten, E.P.3
-
14
-
-
77953501756
-
Health economics in haemophilia: a review from the clinician's perspective
-
Escobar MA. Health economics in haemophilia: a review from the clinician's perspective. Haemophilia. 2010; 16(Suppl 3):29-34.
-
(2010)
Haemophilia.
, vol.16
, pp. 29-34
-
-
Escobar, M.A.1
-
15
-
-
79953306825
-
A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study)
-
Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost. 2011; 9:700-710.
-
(2011)
J Thromb Haemost.
, vol.9
, pp. 700-710
-
-
Gringeri, A.1
Lundin, B.2
von Mackensen, S.3
Mantovani, L.4
Mannucci, P.M.5
-
16
-
-
84870988601
-
A survey of the outcome of prophylaxis, on-demand treatment or combined treatment in 18-35-year old men with severe haemophilia in six countries
-
Noone D, O'Mahony B, van Dijk JP, Prihodova L. A survey of the outcome of prophylaxis, on-demand treatment or combined treatment in 18-35-year old men with severe haemophilia in six countries. Haemophilia. 2013; 19:44-50.
-
(2013)
Haemophilia.
, vol.19
, pp. 44-50
-
-
Noone, D.1
O'Mahony, B.2
van Dijk, J.P.3
Prihodova, L.4
-
19
-
-
65449144703
-
Replacement therapy for invasive procedures in patients with haemophilia: literature review, European survey and recommendations
-
Hermans C, Altisent C, Batorova A, et al. Replacement therapy for invasive procedures in patients with haemophilia: literature review, European survey and recommendations. Haemophilia. 2009; 15:639-658.
-
(2009)
Haemophilia.
, vol.15
, pp. 639-658
-
-
Hermans, C.1
Altisent, C.2
Batorova, A.3
-
20
-
-
59849093369
-
Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A
-
Collins PW, Blanchette VS, Fischer K, et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost. 2009; 7:413-420.
-
(2009)
J Thromb Haemost.
, vol.7
, pp. 413-420
-
-
Collins, P.W.1
Blanchette, V.S.2
Fischer, K.3
-
21
-
-
84859197329
-
Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients
-
Powell JS, Josephson NC, Quon D, et al. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood. 2012; 119:3031-3037.
-
(2012)
Blood.
, vol.119
, pp. 3031-3037
-
-
Powell, J.S.1
Josephson, N.C.2
Quon, D.3
-
22
-
-
84893123337
-
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
-
Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014; 123:317-325.
-
(2014)
Blood.
, vol.123
, pp. 317-325
-
-
Mahlangu, J.1
Powell, J.S.2
Ragni, M.V.3
-
23
-
-
70349229281
-
Pharmacokinetics and dose requirements of factor VIII over the age range 3-74 years: a population analysis based on 50 patients with long-term prophylactic treatment for haemophilia A
-
Bjorkman S, Folkesson A, Jonsson S. Pharmacokinetics and dose requirements of factor VIII over the age range 3-74 years: a population analysis based on 50 patients with long-term prophylactic treatment for haemophilia A. Eur J Clin Pharmacol. 2009; 65:989-998.
-
(2009)
Eur J Clin Pharmacol.
, vol.65
, pp. 989-998
-
-
Bjorkman, S.1
Folkesson, A.2
Jonsson, S.3
-
24
-
-
70350216170
-
Population pharmacokinetics of recombinant factor VIII:C (ReFacto) in adult HIV-negative and HIV-positive haemophilia patients
-
Karafoulidou A, Suarez E, Anastasopoulou I, et al. Population pharmacokinetics of recombinant factor VIII:C (ReFacto) in adult HIV-negative and HIV-positive haemophilia patients. Eur J Clin Pharmacol. 2009; 65:1121-1130.
-
(2009)
Eur J Clin Pharmacol.
, vol.65
, pp. 1121-1130
-
-
Karafoulidou, A.1
Suarez, E.2
Anastasopoulou, I.3
-
25
-
-
77952712296
-
Comparative pharmacokinetics of plasma- and albumin-free recombinant factor VIII in children and adults: the influence of blood sampling schedule on observed age-related differences and implications for dose tailoring
-
Bjorkman S, Blanchette VS, Fischer K, et al. Comparative pharmacokinetics of plasma- and albumin-free recombinant factor VIII in children and adults: the influence of blood sampling schedule on observed age-related differences and implications for dose tailoring. J Thromb Haemost. 2010; 8:730-736.
-
(2010)
J Thromb Haemost.
, vol.8
, pp. 730-736
-
-
Bjorkman, S.1
Blanchette, V.S.2
Fischer, K.3
-
26
-
-
84855853759
-
Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight
-
Bjorkman S, Oh M, Spotts G, et al. Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight. Blood. 2012; 119:612-618.
-
(2012)
Blood.
, vol.119
, pp. 612-618
-
-
Bjorkman, S.1
Oh, M.2
Spotts, G.3
-
27
-
-
84864280808
-
Population pharmacokinetics of plasma-derived factor IX in adult patients with haemophilia B: implications for dosing in prophylaxis
-
Bjorkman S, Ahlen V. Population pharmacokinetics of plasma-derived factor IX in adult patients with haemophilia B: implications for dosing in prophylaxis. Eur J Clin Pharmacol. 2012; 68:969-977.
-
(2012)
Eur J Clin Pharmacol.
, vol.68
, pp. 969-977
-
-
Bjorkman, S.1
Ahlen, V.2
-
28
-
-
0028365342
-
Pharmacokinetics of factor IX in patients with haemophilia B. Methodological aspects and physiological interpretation
-
Bjorkman S, Carlsson M, Berntorp E. Pharmacokinetics of factor IX in patients with haemophilia B. Methodological aspects and physiological interpretation. Eur J Clin Pharmacol. 1994; 46:325-332.
-
(1994)
Eur J Clin Pharmacol.
, vol.46
, pp. 325-332
-
-
Bjorkman, S.1
Carlsson, M.2
Berntorp, E.3
-
29
-
-
0001776145
-
Multidose pharmacokinetics of factor IX: implications for dosing in prophylaxis
-
Carlsson M, Bjorkman S, Berntorp E. Multidose pharmacokinetics of factor IX: implications for dosing in prophylaxis. Haemophilia. 1998; 4:83-88.
-
(1998)
Haemophilia.
, vol.4
, pp. 83-88
-
-
Carlsson, M.1
Bjorkman, S.2
Berntorp, E.3
-
30
-
-
0035216180
-
Ways to fit a PK model with some data below the quantification limit
-
Beal SL. Ways to fit a PK model with some data below the quantification limit. J Pharmacokinet Pharmacodyn. 2001; 28:481-504.
-
(2001)
J Pharmacokinet Pharmacodyn.
, vol.28
, pp. 481-504
-
-
Beal, S.L.1
-
31
-
-
77956756712
-
Erratum to: likelihood based approaches to handling data below the quantification limit using NONMEM VI
-
Ahn JE, Karlsson MO, Dunne A, Ludden TM. Erratum to: likelihood based approaches to handling data below the quantification limit using NONMEM VI. J Pharmacokinet Pharmacodyn. 2010; 37:305-308.
-
(2010)
J Pharmacokinet Pharmacodyn.
, vol.37
, pp. 305-308
-
-
Ahn, J.E.1
Karlsson, M.O.2
Dunne, A.3
Ludden, T.M.4
-
32
-
-
77954851703
-
Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A
-
Bjorkman S. Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A. Haemophilia. 2010; 16:597-605.
-
(2010)
Haemophilia.
, vol.16
, pp. 597-605
-
-
Bjorkman, S.1
-
33
-
-
79951902782
-
Evaluation of the TCIWorks Bayesian computer program for estimation of individual pharmacokinetics of FVIII
-
Bjorkman S. Evaluation of the TCIWorks Bayesian computer program for estimation of individual pharmacokinetics of FVIII. Haemophilia. 2011; 17:e239-e240.
-
(2011)
Haemophilia.
, vol.17
, pp. e239-e240
-
-
Bjorkman, S.1
|