Topical pyridostigmine for ocular myasthenia gravis: a translational hypothesis

Background: Myasthenia gravis (MG) is an autoimmune disorder of the neuromuscular junction predominantly mediated by antibodies that target the acetylcholine receptors in the skeletal muscles. Ocular involvement of the levator palpebrae superioris and extraocular muscles is common, resulting in ptosis and diplopia. Oral pyridostigmine, an acetylcholinesterase, is typically the first-line therapy, but its administration is frequently limited by systemic cholinergic adverse effects and inconsistent relief of ocular symptoms. Hypothesis: animal model ocular permeability and tissue integrity be examined to determine the feasibility of topical pyridostigmine formulation providing effective localized neuromuscular transmission enhancement in patients with ocular MG. Conclusions: animal studies and translational modelling. The feasibility of topical or periocular pyridostigmine as a localized treatment strategy for ocular MG deserves further investigation. If validated, this approach could shift management toward targeted, better-tolerated topical therapies with reduced systemic risk.