Amifampridine phosphate in the treatment of muscle-specific kinase myasthenia gravis: a phase IIb, randomized, double-blind, placebo-controlled, double crossover study.
Silvia BonannoMaria Barbara PasanisiRita FrangiamoreLorenzo MaggiCarlo AntozziFrancesca AndreettaAngela CampanellaGreta BrennaLorenzo CottiniRenato MantegazzaPublished in: SAGE open medicine (2018)
Despite the low number of patients, MuSK-001 study provided evidence that amifampridine phosphate, in the range of 30-60 mg daily dose, was safe and effective in treating muscle-specific kinase myasthenia gravis, suggesting the need for a large multi-center trial to confirm these results.
Keyphrases
- myasthenia gravis
- placebo controlled
- double blind
- phase iii
- phase ii
- clinical trial
- study protocol
- end stage renal disease
- open label
- skeletal muscle
- phase ii study
- chronic kidney disease
- peritoneal dialysis
- protein kinase
- randomized controlled trial
- radiation therapy
- squamous cell carcinoma
- physical activity
- replacement therapy