Compassionate Use of 3,4 Diaminopyridine in Patients with Congenital Myasthenic Syndrome


About this study

This study is being done to find out whether the investigational drug 3,4-DAP will help patients with Congenital Myasthenic Syndrome (CMS), inherited muscle weakness and fatigue.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Patients with documented CMS (based on clinical and electrodiagnostic criteria, and in some cases molecular genetic and microelectrode studies performed on muscle biopsy specimens)

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Eric Sorenson, M.D.

Closed for enrollment

Contact information:

Eric Sorenson M.D.

(507) 284-8729

More information


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