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Dysregulation of TSCs in ALS is thought to lead to impaired and erroneous NMJ recovery and plasticity [290]

In a subsequent phase III RCT with 131 patients with PV, rilzabrutinib failed to show significant differences with regard to remission rates between the drug and placebo arm, although the proportion of participants who achieved the original primary endpoint (complete remission from week 29 to week 37 with a corticosteroid dose of up to 5 mg/day) was initially significantly higher in the rilzabrutinib group [26]