Tag: Neuromuscular Disorders

A phase 3, open-label, single-arm, multicenter study to evaluate the pharmacokinetics, pharmacodynamics, efficacy, safety, and immunogenicity of Ravulizumab administered intravenously in pediatric participants (6 to <18 years of age) with generalized myasthenia gravis (gMG).

A Phase 3, Single-Arm, Multicenter, Open-label Extension of Study ARGX-113-2007 to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy

Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis- ARGX-113-2006. For more information or to register for the study, contact Study Coordinator Manisha Chopra

Brief Summary Long-term, multicenter, multinational, observational, registry of patients with gMG that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT) such as eculizumab (Soliris®) and ravulizumab (Ultomiris®). Detailed Description At the time of enrollment in the Registry, participant records will...

UNC Neurologist, Dr. James F. Howard, was the lead investigator in the clinical trial that led to FDA approval.