3 results
Primary objective of the study is•To investigate the change of Urinary Albumin-to-Creatinine ratio (UCAR) after 90 days treatment Secondary objectives of the study are•To assess safety and tolerability of these doses by assessing the effects on…
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,…
Primary: To assess the efficacy of GNX compared with PBO, as adjunctive therapy for the treatment of primary seizure types in children with genetically-confirmed PCDH19-related epilepsy during the 17-week double-blind (DB) phase. Secondary: • To…