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Safety, Efficacy, and Determination of the Recommended Phase 2 Dose for the Oral Selective Inhibitor of Nuclear Export (SINE) Selinexor (KPT-330)

doi: 10.1126/sciadv.aaw2238 95 DixonSJPatelDNWelschMSkoutaRLeeEDHayanoMet al

The equation F/F 0 = (F-F 0 )/F 0 was used to calculate the fractional fluorescence changes

As SMA can cause a person to have fewer motor neurons in their spinal cords, the spasticity that some people with SMA experience occurs when the muscles are not well-controlled by motor neurons