In multiple sclerosis (MS), macrophages and microglia play a dual role that could be used to treat this neurodegenerative disease. These cells promote inflammation that demyelinates neurons but also ...
– 53.7% mean splicing correction observed following a single 15 mg/kg dose of PGN-EDODM1, with all patients showing an improvement in splicing – – PGN-EDODM1 was generally well-tolerated at 15 mg/kg, ...
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