Gonda 8-104/Joseph 1-242

Muscle
Andrew Engel, M.D.
"Novel Congenital Myasthenias"

Learning objectives:
Summarize the basic principles of neuromuscular transmission
Review general comments on congenital myasthenic syndromes (CMS)
Identify key components of the exocytotic machinery
Identify the safety margin of neuromuscular transmission
Describe CMS targets
Summarize investigation of two recently recognized presynaptic congenital myasthenic syndromes

The presenter (nor spouse/partner) does not have a relevant financial relationship to disclose and does not intend to discuss an off-label/investigative use of a commercial product/device.