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MR6-26-2 is a small molecule drug and an analogue of ebselen, developed as a neuroprotective agent for amyotrophic lateral sclerosis (ALS), particularly familial ALS (fALS) associated with mutations in the SOD1 gene. The compound acts by stabilizing the SOD1 dimer, which is destabilized in both familial and some sporadic ALS cases, leading to toxic protein aggregation. By binding to mutant SOD1 (such as G93A), MR6-26-2 arrests this aggregation and preserves neuromuscular junction integrity, thereby slowing disease onset and progression in preclinical models. Its mechanism has been validated through biochemical assays and co-crystallography studies[1].
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