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Amifampridine

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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

  1. ↑ https://www.neurologylive.com/clinical-focus/amifampridine-phosphate-shows-success-in-myasthenia-gravis-trial
  2. ↑ https://www.drugs.com/news/firdapse-amifampridine-approved-rare-autoimmune-disorder-78751.html

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