One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency.
George A DiazSimon A JonesMaurizio ScarpaKarl Eugen MengelRoberto GiuglianiNathalie GuffonIsabela BatsuPatricia A FraserJing LiQi ZhangCatherine Ortemann-RenonPublished in: Genetics in medicine : official journal of the American College of Medical Genetics (2021)
In this study in children with chronic ASMD, olipudase alfa was generally well-tolerated with significant, comprehensive improvements in disease pathology across a range of clinically relevant endpoints.