Study Description: The purpose of this research study is to allow Dr. Michael Rivner to use amifampridine phosphate (Firdapse) for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS) until the drug is approved for use in the United States by the FDA. Catalyst, the study Sponsor, is paying for the study drug. Presently, there is no FDA-approved treatment available to cure or provide relief from the symptoms of LEMS or CMS.
Principal Investigator: Michael Rivner
Eligibility Criteria: Inclusion Criteria: A confirmed physician diagnosis of LEMS or CMS and completed any anti-cancer treatment for at least 3 months A negative pregnancy test for women who can become pregnant and willingness to use birth control throughtout the study Exclusion Criteria: History of epilepsy, of an unstable heart condition, of kidney or liver impairement, of surviving a near drowning due to loss of consciousness certain CMS subtypes including slow-channel syndrome, LRP4 deficiency, and acetylcholinesterase deficiency.
Compensation: No, subjects will not be compensated.
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