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SMN1

—Survival Motor Neuron 1

Chromosome: 5

Location: 5:70925030-70953942

OMIM: 600354

Ensembl: ENSG00000172062

Official·OMIM:600354 Official·ENSG00000172062

Encodes the SMN protein critical for motor neuron survival. Homozygous deletion/mutation causes SMA.

Treatment Roadmap — SMN1

FDA Approved

Preclinical

Phase 1

1 trial

Phase 2

2 trial

Phase 3

7 trial

Approved

Zolgensma (onasemnogene abeparvovec) — FDA (5/24/2019)

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

Associated Diseases

Spinal Muscular Atrophy

Autosomal RecessivePrevalence: ~60Kneuromuscular

Official·OMIM:253300 Official·ORPHA70

Summary

Clinical Trial

Approved Therapy

Registered Variant

SMN1

Treatment Roadmap — SMN1

Associated Diseases

Summary

Approved Therapies