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autosomal recessive sideroblastic anemia - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

autosomal recessive sideroblastic anemia

MONDO:0016828

Also known as:congenital sideroblastic anaemiacongenital sideroblastic anemiaARSA

Find Clinical TrialsTrials

Overview

Congenital autosomal recessive sideroblastic anemia (ARSA) is a non-syndromic, microcytic/hypochromic sideroblastic anemia, present from early infancy and characterized by severe microcytic anemia, which is not pyridoxine responsive, and increased serum ferritin.

Clinical Trials10 registered · 6 recruiting

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

10 clinical trials registered, 6 recruiting. Interventions under study include drug therapy and other interventions. Pipeline includes 5 PHASE4, 2 PHASE3, 1 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT06884280](https://clinicaltrials.gov/study/NCT06884280)

Data assembled from 4 of 12 sources · Last updated Sep 20, 2026, 3:05 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:260305

GARD

Genetic and Rare Diseases Info Center

GARD:17240

Questions for Your Care Team

→Are any of the 10 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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Research Type	Count	% of Total
Patient case studies	13	48%
Laboratory research	7	26%
Research summaries	5	19%
Other research	1	4%
Disease patterns and progression	1	4%

autosomal recessive sideroblastic anemia

Overview

Research Landscape

Medical References

External Resources

Orphanet

GARD

Subtypes

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Recent Publications