Journal ArticleDOI
Haemophilias A and B.
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TLDR
In the past, men with haemophilia were likely to die in their youth, but with advances in diagnosis, and especially with development of safe and effective treatment, affected individuals can now look forward to a normal life expectancy.About:
This article is published in The Lancet.The article was published on 2003-05-24. It has received 625 citations till now. The article focuses on the topics: Haemophilia & Haemophilia B.read more
Citations
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Journal ArticleDOI
Vascularized Free Tissue Transfer in a Patient with Hemophilia B: Case Report and Literature Review.
TL;DR: A successful case of mandibular reconstruction using a free fibular flap in a patient with severe hemophilia B is reported and the protocols used, as well as a review of the literature of similar cases.
Journal ArticleDOI
Postpolypectomy bleeding secondary to undiagnosed haemophilia B in an octogenarian.
TL;DR: In this case, the patient was operated on as the object did not progress for more days and the attempt for endoscopic removal failed, and the sharpness of the object was also considered.
Journal ArticleDOI
Pain in Hemophilia: Unexplored Role of Oxidative Stress
TL;DR: Cross examine preclinical and clinical studies that address the contribution of oxidative stress in hemophilia and related diseases that affect synovial tissue to induce acute and potentially chronic pain to help provide potential treatable targets using antioxidants to ameliorate pain in Hemophilia.
Journal ArticleDOI
Depression in Family Members of Hemophiliac Patients
TL;DR: The risk for depression increased with the severity of hemophilia, and there is a need to mobilize social resources in order to share the burden of these families and to provide continuous and comprehensive care for family members.
Neonatal Hemophilia A: case Report in Sincelejo (Colombia)
Jorge Mendoza-Herrera,Edward Carvajalino Santiago,Nelson Muñoz Álvarez,Carlos Moneriz Pretell +3 more
TL;DR: This trabajo tiene como objetivo alertar al cuerpo médico sobre the importancia of considerar esta enfermedad dentro del diagnóstico diferencial en los síndromes hemorrágicos del recién nacido, incluso sin the existencia of antecedentes familiares.
References
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Journal ArticleDOI
A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency
Salima Hacein-Bey-Abina,Christof von Kalle,Manfred Schmidt,Françoise Le Deist,NM Wulffraat,Elisabeth McIntyre,Isabelle Radford,Jean-Luc Villeval,Christopher Fraser,Marina Cavazzana-Calvo,Alain Fischer +10 more
TL;DR: The sustained correction of X-linked severe combined immunodeficiency disease by ex vivo, retrovirally mediated transfer of the γc gene into CD34+ cells in four of five patients with the disease has been reported.
Journal ArticleDOI
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
Mark A. Kay,Catherine S. Manno,Catherine S. Manno,Margaret V. Ragni,Peter J. Larson,Peter J. Larson,Linda B. Couto,Alan McClelland,Bertil Glader,Amy J. Chew,Shing J Tai,Roland W. Herzog,Valder R. Arruda,Fred Johnson,Ciaran Scallan,Erik D. Skarsgard,Alan W. Flake,Alan W. Flake,Katherine A. High,Katherine A. High +19 more
TL;DR: Evidence of gene expression at low doses of vector suggests that dose calculations based on animal data may have overestimated the amount of vector required to achieve therapeutic levels in humans, and that the approach offers the possibility of converting severe haemophilia B to a milder form of the disease.
Journal ArticleDOI
Characterization of the human factor VIII gene
Jane Gitschier,William I. Wood,Therese M. Goralka,Karen L. Wion,Ellson Y. Chen,Dennis H. Eaton,Gordon A. Vehar,Daniel J. Capon,Richard M. Lawn +8 more
TL;DR: The complete 186,000 base-pair (bp) human factor VIII gene has been isolated and consists of 26 exons ranging in size from 69 to 3,106 bp and introns as large as 32.4 kilobases as mentioned in this paper.
Journal ArticleDOI
Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B
TL;DR: It appears to be possible to prevent haemophilic arthropathy by giving effective continuous prophylaxis from an early age, and preventing the VIII:C or IX:C concentration from falling below 1% of normal.
Journal ArticleDOI
Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis.
Gilbert C. White,Frits R. Rosendaal,Louis M. Aledort,Jeanne M. Lusher,Chantal Rothschild,Jørgen Ingerslev +5 more
TL;DR: Definitions in Hemophilia - Recommendation of the Scientific Subcommittee on Factor VIII and Factor IX and the Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis.
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