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Amifampridine
Amifampridine phosphate was granted an Orphan Drug designation by the FDA for MG in September 2016, shortly after receiving a Refusal to File letter from the FDA for its submission for the treatment of congenital myasthenic syndromes and Lambert Eaton myasthenic syndrome (LEMS [1])[2]
Links
https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-children-lambert-eaton-myasthenic-syndrome-rare-autoimmune-disorder Drug Trial Snapshot: Firdapse
