A phase 1 study in patients with Fukuyama-type congenital muscular dystrophy
A multicenter phase 1 study of NS-035 in patients with Fukuyama-type congenital muscular dystrophy
Fukuyama-type congenital muscular dystrophy
This study consists of the following 4 cohorts. The study will start with cohort 1 and only D-mannitol will be administered once during the premedication phase, followed by 12 simultaneous doses of NS-035 and D-mannitol once weekly during the treatment phase. The dose of D-mannitol was fixed at 500 mg / kg in all cohorts, and NS-035 was gradually increased from cohort 1 to cohort 4 (1.6 mg/kg, 6.0 mg/kg, 20 mg/kg and 40 mg/kg, respectively). This study consists of the following 4 cohorts. The study will start with cohort 1 and only D-mannit ...