Sickle beta thalassemia is an inherited condition that affects hemoglobin, the protein in red blood cells that carries oxygen to different parts of the body.It is a type of sickle cell disease. Affect...
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 documentation for sickle cell-beta-thalassemia disease syndrome is limited primarily due to its rarity and the complexity of its clinical features. As this condition affects a relatively small population, comprehensive studies and systematic documentation have been challenging. Additionally, the overlapping phenotypes with other hemoglobinopathies complicate the characterization of this syndrome. Ongoing research may provide further insights into its clinical management.
To navigate your care effectively, consider consulting a hematologist with expertise in sickle cell disease and thalassemia. They can provide specialized care tailored to your needs. Additionally, you can explore resources such as the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov for more information and support. Participating in clinical trials may also be an option, and you can find relevant studies through ClinicalTrials.gov. Genetic counseling may be beneficial to understand the implications of this condition for you and your family.
There are currently 15 orphan drugs designated for sickle cell-beta-thalassemia disease syndrome, including 15(S)-hydroxy-(5Z,8Z,11Z,13E,17Z)-eicosapentaenoic acid ethyl ester and rilzabrutinib, among others. Additionally, there are 11 active clinical trials exploring various treatment options. You can find more information about these trials at ClinicalTrials.gov by searching for 'sickle cell-beta-thalassemia disease syndrome'. This research landscape offers hope for future therapies.
Actionable guidance for navigating care for sickle cell-beta-thalassemia disease syndrome
To navigate your care effectively, consider consulting a hematologist with expertise in sickle cell disease and thalassemia. They can provide specialized care tailored to your needs. Additionally, you can explore resources such as the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov for more information and support. Participating in clinical trials may also be an option, and you can find relevant studies through ClinicalTrials.gov. Genetic counseling may be beneficial to understand the implications of this condition for you and your family.
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.
Organizations with approved therapies for this disease
No approved therapies yet
Research is ongoing — 2 companies have orphan drug designations for this disease
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.