An Open-Label, Expanded Access Protocol for Amifampridine Phosphate Treatment in Patients With Congenital Myasthenic Syndrome (CMS)

Investigator: Sheetal Shroff, MD

Study Coordinator:

Status: Open Not Enrolling Number: NCT02189720


Protocol Number: Pro00013058


The primary objective of the study is: • To provide patients with CMS access to amifampridine phosphate therapy until the product becomes commercially available or development is discontinued. The secondary objective of the study is: • To assess the long-term safety of amifampridine phosphate in patients with CMS
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