2 results
Approved WMOCompleted
Primary:* To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.Secondary:* To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study,…
Approved WMORecruiting
Study Objectives:The purpose of this Phase 2 study is to evaluate the safety, efficacy, pharmacokinetics (PK),pharmacodynamics (PD), and immunogenicity of narsoplimab in paediatric-aged patients withthrombotic microangiopathies (TMA) following…