MuSK-Myasthenia gravis (MuSK-MG) is a subclass of the disease characterized by female predominance, early onset, prominent bulbar involvement, severe clinical course, presence of anti MuSK antibodies, and significant resistance to treatment, particularly to cholinesterase inhibitors. An unmet medical need is to improve the therapeutic armamentarium in MuSK-MG. Amifampridine phosphate is already marketed in Europe as first-line symptomatic treatment for LEMS and some of the congenital myasthenic syndromes.

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