Hemophilia B

Common Name(s)

Hemophilia B, Hereditary factor IX deficiency disease, Christmas disease

Hemophilia B is a bleeding disorder that slows the blood clotting process. People with this disorder experience prolonged bleeding or oozing following an injury or surgery. In severe cases of hemophilia, heavy bleeding occurs after minor trauma or even in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. Milder forms may not become apparent until abnormal bleeding occurs following surgery or a serious injury. People with an unusual form of hemophilia B, known as hemophilia B Leyden, experience episodes of excessive bleeding in childhood but have few bleeding problems after puberty. Hemophilia B is inherited in an X-linked recessive pattern and is caused by mutations in the F9 gene.
 

Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Hemophilia B" for support, advocacy or research.

Bleeding Disorders Alliance Illinois

The Bleeding Disorders Alliance Illinois exists to improve the quality of life for persons affected by hemophilia and other inherited bleeding disorders.

Last Updated: 25 Sep 2013

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Hemophilia Federation of America

The Hemophilia Federation of America is a national non-profit organization that assists and advocates for the bleeding disorders community.

Last Updated: 6 Jul 2015

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General Support Organizations

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How do you compare to others with this condition?

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Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Hemophilia B" for support, advocacy or research.

Bleeding Disorders Alliance Illinois

The Bleeding Disorders Alliance Illinois exists to improve the quality of life for persons affected by hemophilia and other inherited bleeding disorders.

http://www.bdai.org

Last Updated: 25 Sep 2013

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Hemophilia Federation of America

The Hemophilia Federation of America is a national non-profit organization that assists and advocates for the bleeding disorders community.

http://www.hemophiliafed.org

Last Updated: 6 Jul 2015

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General Support Organizations

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Scientific Literature

Articles from the PubMed Database

Research articles describe the outcome of a single study. They are the published results of original research.
The terms "Hemophilia B" returned 186 free, full-text research articles on human participants. First 3 results:

[The skin as a vehicle for gene therapy: hemophilia B, an application model].
 

Author(s): Isaura Araceli González-Ramos, Ana Rebeca Jaloma-Cruz

Journal: Gac Med Mex. ;151(2):266-9.

 

Artificial skin offers important advantages in gene therapy tor its biosafety and simple monitoring. An easy access of keratinocytes through small biopsies and their in vitro expansion enriched with epithelial stem cells, make them an ideal target for long-term therapeutic transgene ...

Last Updated: 7 May 2015

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A natural choice for hemophilia B.
 

Author(s): Dwight D Koeberl

Journal: Blood. 2015 Mar;125(10):1509-10.

 

In this issue of Blood, Crudele et al describe a novel study of adeno-associated virus (AAV) vector-mediated gene therapy that induced immune tolerance to factor IX (FIX) in a hemophilia B (HB) dog with previously formed anti-FIX inhibitor antibodies (IAs).

Last Updated: 6 Mar 2015

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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.
 

Author(s): Julie M Crudele, Jonathan D Finn, Joshua I Siner, Nicholas B Martin, Glenn P Niemeyer, Shangzhen Zhou, Federico Mingozzi, Clinton D Lothrop, Valder R Arruda

Journal: Blood. 2015 Mar;125(10):1553-61.

 

Emerging successful clinical data on gene therapy using adeno-associated viral (AAV) vector for hemophilia B (HB) showed that the risk of cellular immune response to vector capsid is clearly dose dependent. To decrease the vector dose, we explored AAV-8 (1-3 × 10(12) vg/kg) encoding ...

Last Updated: 6 Mar 2015

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Reviews from the PubMed Database

Review articles summarize what is currently known about a disease. They discuss research previously published by others.
The terms "Hemophilia B" returned 5 free, full-text review articles on human participants. First 3 results:

Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.
 

Author(s): Timothy C Nichols, Margaret H Whitford, Valder R Arruda, Hansell H Stedman, Mark A Kay, Katherine A High

Journal: Hum Gene Ther Clin Dev. 2015 Mar;26(1):5-14.

 

Preclinical testing of new therapeutic strategies in relevant animal models is an essential part of drug development. The choice of animal models of disease that are used in these studies is driven by the strength of the translational data for informing about safety, efficacy, and ...

Last Updated: 17 Mar 2015

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Consensus review of the treatment of cardiovascular disease in people with hemophilia A and B.
 

Author(s): Victor A Ferraris, Leonard I Boral, Alice J Cohen, Susan S Smyth, Gilbert C White

Journal: Cardiol Rev. ;23(2):53-68.

 

With advances in care, increasing numbers of people with hemophilia (PWH) achieve near-normal life expectancies and present with typical age-related cardiovascular conditions. Evidence-based guidelines for medical or surgical management of cardiovascular conditions in individuals ...

Last Updated: 6 Feb 2015

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Hemophilia A and hemophilia B: focus on arthropathy and variables affecting bleeding severity and prophylaxis.
 

Author(s): M Escobar, S Sallah

Journal: J. Thromb. Haemost.. 2013 Aug;11(8):1449-53.

 

Hemophilia A (HA) and hemophilia B (HB) are X-linked, recessive disorders. Although their clinical manifestations are essentially indistinguishable, it has been suggested that bleeding episodes in patients with HA are generally more severe and occur at higher frequency than in patients ...

Last Updated: 15 Aug 2013

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Symptoms, Diagnosis, and Treatment

There are currently no related results available in Genetics Home Reference.

 
 
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Clinical Trial Information This information is provided by ClinicalTrials.gov

A Gene Therapy Study for Hemophilia B
 

Status: Recruiting

Condition Summary: Hemophilia B

 

Last Updated: 26 Feb 2016

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Last Updated: 23 Feb 2016

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Last Updated: 17 Feb 2016

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