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FMR1

—Fragile X Messenger Ribonucleoprotein 1

Chromosome: X

Location: X:147911858-147951125

OMIM: 309550

Ensembl: ENSG00000102081

Official·OMIM:309550 Official·ENSG00000102081

Encodes FMRP protein. CGG repeat expansion causes Fragile X syndrome.

Treatment Roadmap — FMR1

Preclinical

Phase 1

Phase 2

2 trial

Phase 3

1 trial

Approved

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

Associated Diseases

Fragile X Syndrome

X-Linked DominantPrevalence: ~1Mneurological

Official·OMIM:300624 Official·ORPHA908

Summary

Clinical Trial

Approved Therapy

Registered Variant