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Myasthenic Syndromes, Congenital clinical trials at UC Health
1 research study open to new patients

  • 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

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