A rare, genetic hemoglobinopathy characterized by all the characteristics of sickle cell anemia (SCA). Clinical course is similar to SCA, including acute episodes of pain, splenic infarction and splen...
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 Feb 5, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 5, 2026
Connect with organizations supporting the sickle cell-hemoglobin d disease syndrome community
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
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 Feb 5, 2026
SCDAA is the premier organization advocating for people with sickle cell disease.
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. 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.
AI-curated news mentioning sickle cell-hemoglobin d disease syndrome
Updated Feb 6, 2026
Yale School of Medicine discusses the potential of gene-editing treatments for rare genetic diseases, highlighting the FDA's recent approval of a CRISPR-based therapy for sickle cell disease in 2023. This marks a significant advancement in the application of CRISPR technology.
The S 735 bill, aimed at enhancing research, surveillance, prevention, and treatment for sickle cell disease and other heritable blood disorders, has been introduced and referred to the Committee on Health, Education, Labor, and Pensions. This legislative move could impact funding and focus on these conditions.
The House has introduced HR 1796, aimed at enhancing research, surveillance, prevention, and treatment for sickle cell disease and other heritable blood disorders. The bill has been referred to the House Committee on Energy and Commerce for further consideration.
The Federal Register has published updates regarding the Sickle Cell Advisory Committee, including amendments and renewals. This committee plays a crucial role in advising on policies related to sickle cell disease.
NHS Scotland has approved Exagamglogene autotemcel, a CRISPR gene therapy for severe sickle cell disease, promising durable reduction in vaso-occlusive crises. This approval is contingent on a Patient Access Scheme, allowing for confidential pricing arrangements for this high-cost therapy.