Disease

Spinal muscular atrophy-11

Gene

SMN1

Inheritance AR
Current Treatments risdiplam , nusinersen, gene therapy (onasemnogene abeparvovec-xioi)
Evidence for Treatment guideline2, 3
Non-Molecular Testing Availability yes
Non-Molecular Test electromyography and nerve conduction studies 4
Disease Frequency (per 100,000) 105
Genes Included in Disease Frequency SMN1
Experimental / Investigational Therapies https://clinicaltrials.gov/ct2/results?cond=Spinal+Muscular+Atrophy&term=&cntry=&state=&city=&dist=
Last Updated: March 5, 2026
RX Genes
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