Severe hemophilia B is a form of hemophilia B characterized by a large deficiency of factor IX leading to frequent spontaneous hemorrhage and abnormal bleeding as a result of minor injuries, or follow...
Comprehensive, easy-to-understand information about this condition
How we create this content →Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
The limited documentation surrounding severe hemophilia B can be frustrating. This condition affects fewer than 10 individuals per million, making it difficult to conduct large-scale clinical studies. Furthermore, the absence of identified genetic factors and systematic documentation of clinical features contributes to the scarcity of information. However, ongoing research efforts aim to improve our understanding and management of this rare disorder.
To navigate your care effectively, consider consulting a hematologist with expertise in bleeding disorders, particularly hemophilia. They can provide tailored management strategies and monitor your condition. Additionally, you may want to explore participation in clinical trials, which can offer access to cutting-edge treatments. Resources like the National Organization for Rare Disorders (NORD) at rarediseases.org can provide support and information about ongoing research and potential clinical trials. Genetic counseling may also be beneficial, especially if family planning is a consideration.
There are several orphan drugs designated for severe hemophilia B, including Coagulation Factor IX (recombinant), concizumab-mtci, etranacogene dezaparvovec-drlb, fidanacogene elaparvovec-dzkt, and fitusiran. Additionally, 13 active clinical trials are currently exploring various treatment options. For more information on these trials, you can visit ClinicalTrials.gov and search for 'severe hemophilia B'. This research landscape provides hope for improved therapies and management strategies in the future.
Actionable guidance for navigating care for severe hemophilia B
To navigate your care effectively, consider consulting a hematologist with expertise in bleeding disorders, particularly hemophilia. They can provide tailored management strategies and monitor your condition. Additionally, you may want to explore participation in clinical trials, which can offer access to cutting-edge treatments. Resources like the National Organization for Rare Disorders (NORD) at rarediseases.org can provide support and information about ongoing research and potential clinical trials. Genetic counseling may also be beneficial, especially if family planning is a consideration.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Consider asking your healthcare providers these condition-specific questions
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.