Catalyst Pharmaceutical Partners, Inc – Congenital myasthenic syndrome with episodic apnea

Submitted By:
[email protected]
Published Date:
January 25, 2020

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<p><strong>The primary objective of the study is:&nbsp;</strong></p>
<p>To provide patients with LEMS/CMS/downbeat nystagmus access to amifampridine phosphate therapy where physicians would like to treat with amifampridine phosphate</p>
<p><strong>The secondary objective of the study is:&nbsp;</strong></p>
<p>To assess the long-term safety of amifampridine phosphate in patients with LEMS/CMS/downbeat nystagmus</p>

Rare Disease:
Congenital myasthenic syndrome with episodic apnea