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Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

Bakri Elsheikh · 2021
ClinicalTrials.gov · Datasets · License: Public Domain · 2021
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spinal-muscular-atrophy
This is an observational study to investigate the improvement of NMJ defects in adult patients with SMA following treatment with Risdiplam. Eligible patients will have received treatment with daily oral Risdiplam after receiving approval through their commercial insurance or drug assistance program. All subjects will be evaluated at one visit. Eligible subjects must have been receiving risdiplam for at least 12 months.
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