3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)

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.

Sex

All

Minimum age

2 Years

Inclusion Criteria:

* Diagnosis of LEMS or CM
* If female and over the age of 9, must have a negative pregnancy test, and, if premenopausal, must be willing to practice an effective form of birth control.
* Must be tested and found by ECG not to have a prolonged Q-Tc syndrome.
* Must agree to have a second ECG at the time of peak drug effect.

Exclusion Criteria:

* Known to have sensitivity to 3,4-DAP
* History of clinical seizures or evidence of seizure activity on screening EEG
* History of severe asthma