Results 331 to 340 of about 21,171,014 (388)

Recombinant factor VIII products and inhibitor development in previously untreated patients with severe haemophilia A: Combined analysis of three studies

Haemophilia, 2019
Standard treatment of congenital haemophilia A is based on replacement therapy with coagulation factor VIII (FVIII) products. A major complication of FVIII therapy is the occurrence of IgG alloantibodies (inhibitors) that neutralize FVIII activity.
P. Volkers, K. Hanschmann, T. Calvez, H. Chambost, P. Collins, V. Demiguel, D. Hart, C. Hay, J. Goudemand, R. Ljung, B. Palmer, E. Santagostino, Ella M van Hardeveld, M. Van den berg, B. Keller-Stanislawski   +14 more
semanticscholar   +1 more source

Multiple bleeds in haemophilia A

Clinical & Laboratory Haematology, 2008
One hundred and eighty-one bleeding episodes involving two sites simultaneously were noted during a survey of 4935 bleeding episodes, an incidence of 3.7%. Elbows, knees and ankles were the commonest sites involved in double bleeds, while the thigh, upper arm and elbow were the commonest sites involved in the double bleeds needing most transfusions ...
A, Aronstam, M J, Painter, J V, Eddey
openaire   +2 more sources

Molecular Genetics in Haemophilia A

Vox Sanguinis, 2000
Efficient mutation screening methods have greatly facilitated the analysis of the factor VIII gene. The fast growing number of identified mutations has led to an increasing understanding of the genetics in haemophilia A. In combination with the recently generated molecular models of the factor VIII protein systematic studies of structural‐functional ...
J, Oldenburg, H H, Brackmann, P, Hanfland, R, Schwaab   +3 more
openaire   +2 more sources

Classical haemophilia in a girl

Blut, 1979
A three-year-old white girl from a haemophiliac A family was a symptomatic carrier. The clinical and laboratory data concur with the form of a heterozygous symptomatic carrier state.
A, Dvilansky, I, Nathan, A, Eldor
openaire   +2 more sources

Haemophilia B- in a girl

Clinical & Laboratory Haematology, 2008
Haemophilia B is extremely rare in females and so far 20 cases have been reported. A 9-year-old girl with severe haemophilia symptoms is described, who shows a very low level of factor IX activity (1.5%) and antigen (less than 10%), normal XX female karyotype and negative family history of bleeding tendency or consanguinity.
A, Yoshioka, S, Mikami, Y, Fujimura, T, Sakai, H, Fukui   +4 more
openaire   +2 more sources

Immunologic Studies in Haemophilia A

Scandinavian Journal of Haematology, 1973
The defect in haemophilia A may be due to the absence or deficiency of factor VIII or to the formation of a defective factor VIII. With the use of both an inhibitor neutralising test and a heterologous antiserum against factor VIII 44 patients with haemophilia A were studied for factor VIII related antigenic material in plasma.
L, Holmberg, I M, Nilsson
openaire   +2 more sources

Haemophilia A and Haemophilia B

2019
WOS ...
Carlos Rodriguez-Merchan, E., Hoots, Keith, Tuddenham, Edward, Ragni, Margaret, Hedner, Ulla, DiMichele, Donna M., Lusher, Jeanne, Konkle, Barbara, Brand, Brigit, Escobar, Miguel A., Ingerslev, Jorgen, Kessler, Craig M., Marbet, German A., Schulman, Sam, Marder, Victor, Beeton, Karen, Abshire, Tom, Kulkarni, Roshni, Preston, Francis E., Kavakli, Kaan, Berntorp, Erik   +20 more
openaire   +1 more source

Molecular basis of haemophilia A

Haemophilia, 2004
Summary.  Technologies in molecular biology have greatly advanced the knowledge regarding the origin of haemophilia A and the physiology of the factor VIII (FVIII) protein. A variety of different mutations in the FVIII gene have been identified and their effects on the FVIII protein described.
J, Oldenburg, N M, Ananyeva, E L, Saenko
openaire   +2 more sources

Comparison of the efficacy and safety of 12‐month low‐dose factor VIII tertiary prophylaxis vs on‐demand treatment in severe haemophilia A children

Haemophilia, 2019
Prophylaxis has commonly become standard treatment for severe haemophilia patients. The World Federation of Hemophilia (WFH) recommends low‐dose prophylaxis in countries with resource constraints.
N. Chozie, Fitri Primacakti, D. Gatot, Rahajuningsih D. Setiabudhy, A. Tulaar, Marcel Prasetyo   +5 more
semanticscholar   +1 more source

RNA repair for haemophilia A

Expert Reviews in Molecular Medicine, 2006
The mainstay of gene transfer studies is the use of wild-type cDNAs to effect phenotypic correction of diseases. However, this strategy is not feasible for genetic diseases caused either by mutations of large genes or by dominant-negative mutations, or where the regulation of the gene is critical.
Hengjun, Chao, Christopher E, Walsh
openaire   +2 more sources