Ricardo Maselli – Lambert-Eaton Myasthenic Syndrome

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Congenital myasthenia and LEMS are potentially lethal disorder, which, even with careful
management, significantly impedes participation in normal daily functions. Currently
approved therapies have had little impact on promoting a normal quality of life activity in
these patients. The goal is to systematically examine the effect of 3,4-DAP on the natural
course of this disease and to gain additional experience in titrating 3,4-DAP with other
available therapies to maximize clinical function and development in this patient

The specific aim of this study is to evaluate the use of 3,4 Diaminopyridine (DAP) on
selected patients proven by genetic or serum antibody testing to have Congenital Myasthenic
Syndrome (CMS) or Lambert-Eaton Myasthenic Syndrome (LEMS). We will evaluate the patient for
CMS or LEMS, prescribe 3,4 DAP, and then clinically evaluate the response.