Lambert-Eaton syndrome is an ultra-rare presynaptic myasthenic syndrome of autoimmune origin. A European registry has been established with the support of the pharmaceutical industry and has identified 96 patients followed in 30 clinical centres:
- three-quarters of them were receiving specific treatment in the form of 3-4-DAP (3-4 didydro-aminopyridine) or one of its derivatives, 3-4 DAPP,
- a quarter had additional adjuvant treatment (azathioprine, corticosteroids, pyridostigmine and/or immunoglobulins),
- 30 participants in the registry had cerebellar ataxia of uncertain significance,
- the vast majority of patients remained stable over time.
The registry provides information on the natural history of the syndrome and on the tolerability, judged to be very good overall, of therapies such as 3-4-DAP.