Tessa L Marburger – Congenital Myasthenia (CM)

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Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder which affects the
nerve-muscle junction. The major symptoms of LEMS are progressive muscle weakness. Many
patients experience other symptoms like dry mouth or impotence. Congenital Myasthenia (CM)
is an inherited disorder with similar affects and symptoms.

3,4-Diaminopyridine (DAP) is an experimental drug that has improved strength in some
subjects with (LEMS). There are no other accepted treatments for LEMS and DAP has relatively
few side effects.

Rare Disease: