Results 81 to 90 of about 32,062 (289)

Enhancing the Evidence for Care in Underserved Bleeding Disorders Communities

Haemophilia, EarlyView.
ABSTRACT Background Major advances in haemophilia care have not translated equitably across all populations. Individuals with rare bleeding disorders (RBDs), people living in low‐ and lower‐middle‐income countries (LMICs) and women and girls with inherited bleeding disorders (WGWBD) continue to face significant diagnostic, therapeutic and research ...
Johnny Mahlangu
wiley   +1 more source

Living with Haemophilia, [PDF]

Archives of Disease in Childhood, 1999
Living with Haemophilia, 4th ed. By Jones P. (Pp 412; paperback £14.99; hardback £26.95.) Oxford: Oxford University Press, 1995. ISBN 0 192 62961 1 paperback; 0 192 63030 X hardback. The diagnosis of haemophilia in a newborn son often leaves parents feeling completely overwhelmed.
openaire   +2 more sources

Severe Recurrent Achalasia Cardia Responding to Treatment of Severe Autoimmune Acquired Haemophilia

Case Reports in Gastroenterology, 2012
Acquired haemophilia A and severe acquired achalasia are both very rare conditions with unknown aetiology. Haemophilia A is a haemorrhagic disease induced by deficiency or malfunction of coagulation factor VIII.
H. Al-Jafar, M. Laffan, S. Al-Sabah, M. Elmorsi, M. Habeeb, F. Alnajar   +5 more
doaj   +1 more source

Factor Viii Inhibitors In Patients With Congenital Severe Haemophilia A And Its Relation To Genotype

, 2015
[No abstract available]186e411e414Graw, J., Brackmann, H.H., Oldenburg, J., Schneppenheim, R., Spannagl, M., Schwaab, R., Haemophilia A: from mutation analysis to new therapies (2005) Nat Rev Genet, 6, pp.
Ozelo M.C., Pio S.F., de carvalho B.V., Oliveira G.C., Caram C., Zouain D., dos Santos A., Rezende S.M.   +7 more
core   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

A comparative analysis of routine hospital admission data for sickle cell anaemia, cystic fibrosis and haemophilia in England

Scientific Reports
Recent evidence of inequalities in healthcare for sickle cell disease in the UK is lacking. We aimed to describe trends in hospital admissions for sickle cell anaemia (SCA), cystic fibrosis (CF) and haemophilia in England.
Rutendo Muzambi, Alex Bottle, Mark Layton, Daniel Dexter, Siobhán B. Carr, Carl Reynolds, John James, Cherelle Augustine, Ganesh Sathyamoorthy, Frédéric B. Piel   +9 more
doaj   +1 more source

FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives

Haemophilia, EarlyView.
ABSTRACT Emicizumab has revolutionized hemophilia A care, yet limitations regarding the “ceiling” of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next‐
Tadashi Matsushita, Keiji Nogami, Chandrakala Shanmukhaiah   +2 more
wiley   +1 more source

N‐Methyl‐3,4‐methylendioxymethamphetamine (MDMA)‐related coagulopathy and rhabdomyolysis: A case series and literature review

Research and Practice in Thrombosis and Haemostasis, 2020
Coagulation changes, thrombosis, and hemorrhage have been described in patients following N‐methyl‐3,4‐methylenedioxymethylamphetamine (MDMA) intoxication who subsequently developed serotonin syndrome and rhabdomyolysis.
Andrew J. Doyle, Joel Meyer, Karen Breen, Beverley J. Hunt   +3 more
doaj   +1 more source

Quality of life in adult patients with haemophilia - a single centre experience from Sweden

, 2012
Summary: Increased or maintained health and quality of life (HRQoL) are essential goals in health care among patients with a chronic disease. To gain an understanding of HRQoL in patients with haemophilia at the Haemophilia Treatment Centre in Malmö ...
Lindvall, Karin,, Berntorp, Erik,, Lund University., Von Mackensen, S   +3 more
core   +1 more source

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source