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ADA

—Adenosine Deaminase

Chromosome: 20

Location: 20:44584896-44652252

OMIM: 608958

Ensembl: ENSG00000196839

Official·OMIM:608958 Official·ENSG00000196839

Encodes adenosine deaminase in purine metabolism. Deficiency causes severe combined immunodeficiency.

Treatment Roadmap — ADA

EMA Approved

Preclinical

Phase 1

45 trial

Phase 2

28 trial

Phase 3

37 trial

Approved

Strimvelis — EMA (5/26/2016)

Clinical trials have approximately 10% success rate. This roadmap shows the current status, not guaranteed outcomes.

Associated Diseases

ADA-SCID

Autosomal RecessivePrevalence: ~500immunologic

Official·OMIM:102700 Official·ORPHA277

Summary

168

Clinical Trial

Approved Therapy

Registered Variant

ADA

Treatment Roadmap — ADA

Associated Diseases

Summary

Approved Therapies