• Congenital Myasthenic Syndromes Trials

    Title: Open Label Trial of 3,4 Diaminopyridine in Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS)

    Study Identifier: LCID:2001-040; 3, 4 DAP study

    Principal Investigator: Jayashri Srinivasan, MD, PhD 

    Sponsor: Lahey Hospital & Medical Center 

    Purpose: This study is looking at the effectiveness and adverse effects of 3, 4 Diaminopyridine (3, 4 DAP) for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS).  

    Summary: Options for treating Congenital Myasthenic Syndromes (CMS) are limited.

    Congenital or hereditary myasthenic syndromes (CMS) are neuromuscular disorders caused by genetic defects. There are different subtypes, each caused by a specific genetic mutation. All CMS are characterized by skeletal muscle weakness that varies in severity. Weakness of the ocular (eye), bulbar (mouth and throat), facial, trunk or limb muscles are the most commonly affected muscle groups.

    The drug, 3, 4 Diaminopyridine (3, 4 DAP) has been demonstrated to be effective in treating weakness associated with LEMS and CMS. This study is an open-label study (meaning all study participants) receive 3,4 DAP.

    Study Type: Investigational Drug- 3,4 Diaminopyridine (3,4DAP) 
    Length/Duration: The study consists of monthly clinic visits for the first 4 months. After 4 months, clinic visits will occur every 6 months.  Treatment will be continued indefinitely if a good clinical response is achieved and side effects are tolerated. 


    ELIGIBILITY: If you are interested in this study and would like to see if you may qualify, please complete a brief eligibility form.  

    Contact: For more information on this study, please contact the study coordinator at Jordan.Jara@lahey.org or (781) 744-2958

    Clinical Trials.gov Identifier:  NCT00872950

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