[HTML][HTML] Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome
CJ Hendriksz, R Parini, MD AlSayed, J Raiman… - Molecular genetics and …, 2016 - Elsevier
Long-term efficacy and safety of elosulfase alfa enzyme replacement therapy were
evaluated in Morquio A patients over 96 weeks (reaching 120 weeks in total from pre-
treatment baseline) in an open-label, multi-center, phase III extension study. During this
extension of a 24-week placebo-controlled phase III study, all patients initially received 2.0
mg/kg elosulfase alfa either weekly or every other week, prior to establishment of 2.0
mg/kg/week as the recommended dose, at which point all patients received weekly …
evaluated in Morquio A patients over 96 weeks (reaching 120 weeks in total from pre-
treatment baseline) in an open-label, multi-center, phase III extension study. During this
extension of a 24-week placebo-controlled phase III study, all patients initially received 2.0
mg/kg elosulfase alfa either weekly or every other week, prior to establishment of 2.0
mg/kg/week as the recommended dose, at which point all patients received weekly …