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An Open-Label, Expanded Access Protocol for Firdapse® (Amifampridine Phosphate; 3,4-Diaminopyridine Phosphate) Treatment in Pediatric Patients With Lambert-Eaton Myasthenic Syndrome (LEMS), and in Pediatric and Adult Patients With Congenital Myasthenic Syndromes (CMSs)

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Trial Profile

An Open-Label, Expanded Access Protocol for Firdapse® (Amifampridine Phosphate; 3,4-Diaminopyridine Phosphate) Treatment in Pediatric Patients With Lambert-Eaton Myasthenic Syndrome (LEMS), and in Pediatric and Adult Patients With Congenital Myasthenic Syndromes (CMSs)

Status: Completed
Phase of Trial: Clinical Phase Unknown

Latest Information Update: 31 Mar 2025

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At a glance

  • Drugs Amifampridine (Primary)
  • Indications Congenital myasthenic syndromes
  • Focus Expanded access; Therapeutic Use
  • Sponsors Catalyst Pharmaceuticals

Most Recent Events

  • 05 Dec 2019 Status changed from recruiting to completed.
  • 27 May 2014 New trial record
  • 23 Apr 2014 Programme enrolment will begin in the next few months, pending US FDA review and approvals, according to a Catalyst Pharmaceutical Partners media release.

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