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ABCA4

—ATP Binding Cassette Subfamily A Member 4

Chromosome: 1

Location: 1:93992834-94121177

OMIM: 601691

Ensembl: ENSG00000198691

Official·OMIM:601691 Official·ENSG00000198691

Encodes retinal ABC transporter. Mutations cause Stargardt disease.

Treatment Roadmap — ABCA4

Preclinical

Phase 1

Phase 2

1 trial

Phase 3

Approved

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

Associated Diseases

Stargardt Disease

Autosomal RecessivePrevalence: 1/10000ophthalmologic

Official·OMIM:248200 Official·ORPHA827

Summary

Clinical Trial

Approved Therapy

Registered Variant