Results 1 to 10 of about 21,171,014 (388)

The profile of patients with haemophilia managed at a haemophilia treatment centre in Pretoria, Gauteng

South African Family Practice, 2022
Background: Haemophilia A and B are X-linked recessive bleeding disorders resulting from a deficiency of factors VIII and IX, respectively. Early diagnosis and a comprehensive approach to management is mandatory.
Lethukuthula Mafisa, Abegail N. Dlova, Vanessa Moodley   +2 more
doaj   +3 more sources

Haemophilia [PDF]

, 2011
Although the nature of haemophilia has been understood for thousands of years, knowledge of its molecular genetics is recent. These X-linked bleeding disorders have diverse underlying DNA defects and, in 1992, DNA inversion within the X chromosome was ...
Cahill, Mary R., Colvin, B. T.
core   +3 more sources

Journeying Towards Excellence in the Care of Patients with Haemophilia and Other Inherited Bleeding Disorders From a Developing World: Insights From Tanzania [PDF]

Journal of Blood Medicine
Stella Samson Rwezaula,1– 3 Samson D Mtoba,2 Rashid A Gosse,2 Rebecca W Mwakichako,2 Abdallah Raphael Makalla,1 Martha Loiseyeki Bruckman,1 Luhongedzo Gerson Matandala,1 Linda Alphey Shao,1 Hedwiga Francis Swai,1 Michelle Sholzberg,4 Jerome Teitel,4 Amos
Rwezaula SS, Mtoba SD, Gosse RA, Mwakichako RW, Makalla AR, Bruckman ML, Matandala LG, Shao LA, Swai HF, Sholzberg M, Teitel J, Mwakigonja AR, Akinloye O   +12 more
doaj   +2 more sources

ACQUIRED HAEMOPHILIA A

Journal of Ayub Medical College Abbottabad, 2023
Acquired haemophilia A (AHA) is a rare disease believed to be caused by spontaneous inhibition of clotting Factor VIII by autoantibodies. This is in contrast to the more common congenital haemophilias which are largely due to an absolute deficiency in coagulation factors. It has a prevalence of approximately one per million per year.
Ewe Jin Koh, Jenq Tzong Tan, Nur Nadia Amalina Manan, Khai Yng Hor, Zi Yu Teh, Ming Lee Chin   +5 more
openaire   +2 more sources

Emicizumab prophylaxis in haemophilia A with inhibitors: Three years follow‐up from the UK Haemophilia Centre Doctors’ Organisation (UKHCDO)

Haemophilia, 2023
The UK National Haemophilia Database (NHD) collects data from all UK persons with haemophilia A with inhibitors (PwHA‐I). It is well‐placed to investigate patient selection, clinical outcomes, drug safety and other issues not addressed in clinical trials
C. Wall, H. Xiang, B. Palmer, E. Chalmers, P. Chowdary, P. Collins, Simon Fletcher, G. Hall, D. Hart, M. Mathias, Paul Sartain, S. Shapiro, D. Stephensen, K. Talks, C. Hay   +14 more
semanticscholar   +1 more source

Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Haemophilia, 2023
The standard of care in severe haemophilia A is prophylaxis, which has historically aimed for a factor VIII (FVIII) trough level of ≥1%. However, despite prophylactic treatment, people with haemophilia remain at risk of bleeds that have physical and ...
Lynn Malec, Davide Matino
semanticscholar   +1 more source

Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A

Haemophilia, 2021
Valoctocogene roxaparvovec is an investigational AAV5‐based factor VIII (FVIII) gene therapy that has demonstrated sustained clinical benefit in people with severe haemophilia A.
K. J. Pasi, M. Laffan, S. Rangarajan, T. Robinson, N. Mitchell, W. Lester, E. Symington, B. Madan, Xinqun Yang, Benjamin Kim, Glenn F Pierce, W. Y. Wong   +11 more
semanticscholar   +1 more source

Low immunogenicity of emicizumab in persons with haemophilia A

Haemophilia, 2021
Emicizumab is a humanised, bispecific monoclonal antibody mimicking the cofactor function of activated factor (F)VIII. It is indicated for routine prophylaxis of bleeding episodes in persons with haemophilia A (PwHA) with/without FVIII inhibitors.
C. Schmitt, Thomas Emrich, S. Chebon, E. Fernandez, Claire Petry, K. Yoneyama, A. Kiialainen, Monet Howard, M. Niggli, I. Paz‐Priel, Tiffany Y. Chang   +10 more
semanticscholar   +1 more source

The effect of emicizumab prophylaxis on long‐term, self‐reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies

Haemophilia, 2021
Severe haemophilia A (HA) has a major impact on health‐related quality of life (HRQoL).
M. Skinner, C. Négrier, I. Paz‐Priel, S. Chebon, V. Jiménez‐Yuste, M. Callaghan, M. Lehle, M. Niggli, J. Mahlangu, A. Shapiro, M. Shima, A. Campinha-Bacote, G. Levy, J. Oldenburg, S. von Mackensen, S. Pipe   +15 more
semanticscholar   +1 more source

Gene Therapy in Hemophilia: A Transformational Patient Experience

Journal of Patient Experience, 2023
Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute ...
Enayet Rasul, Ryan Hallock, Magnus Hellmann, Jay Konduros, Luke Pembroke, Gregory LeCleir, Jean Malacan PharmD, Sylvia von Mackensen PhD   +7 more
doaj   +1 more source