3 results
The primary objectives of this study is:* To determine the safety and tolerability of rhHNS via ascending doses administered via a surgically implanted intrathecal drug device (IDDD) once monthly for 6 months, in patientswith MPS IIIA.The secondary…
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in each of the following cohorts: • NMDAR autoimmune encephalitis (AIE) cohort: adults and adolescents (12-16) with definite…
Primary objectiveTo confirm superiority of oral semaglutide at the maximum tolerated dose* (3 mg, 7 mg or 14 mg) versus placebo on glycaemic control in children and adolescents (age 10 to <18 years) with type 2 diabetes on a background…