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Congenital Myasthenic Syndrome clinical trials at UC Health
3 in progress, 1 open to eligible people

  • Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

    open to eligible people ages 2-70

    This randomized, double-blind, controlled, outpatient two-period, two-treatment crossover study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients (ages 2 and above) diagnosed with certain genetic subtypes of CMS and demonstrated open label (amifampridine phosphate) or history of sustained amifampridine benefit from treatment.

    at UCLA

  • Expanded Access Study Amifampridine Phosphate in Congenital Myasthenic Syndrome (CMS)

    Sorry, not accepting new patients

    The primary objective of the study is: • To provide patients with CMSaccess to amifampridine phosphate therapy until the product becomes commercially available or development is discontinued. The secondary objective of the study is: • To assess the long-term safety of amifampridine phosphate in patients with CMS

    at UCLA

  • Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia and Lambert-Eaton Syndrome

    Sorry, not accepting new patients

    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 population. 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.

    at UC Davis

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