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IDUA

—Alpha-L-Iduronidase

Chromosome: 4

Location: 4:986997-1008344

OMIM: 252800

Ensembl: ENSG00000127415

Official·OMIM:252800 Official·ENSG00000127415

Encodes alpha-L-iduronidase enzyme. Mutations cause MPS I (Hurler/Scheie) syndrome.

Treatment Roadmap — IDUA

Preclinical

Phase 1

1 trial

Phase 2

Phase 3

1 trial

Approved

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

Associated Diseases

Mucopolysaccharidosis Type I (Hurler)

Autosomal RecessivePrevalence: 1/100000lysosomal

Official·OMIM:607014 Official·ORPHA579

Summary

Clinical Trial

Approved Therapy

Registered Variant