6 results
To assess the efficacy of idebenone, compared to placebo, in improving or delaying the loss of respiratory function in patients with DMD
The primary objective of the PROTI study is to establish whether patients can correctly determine which treatment assignment they received during the randomised phase of the trial.The key secondary objective is to compare the rate of withdrawal from…
- To gather long-term data on the safety and tolerability of idebenone inFriedreich*s Ataxia- To explore the effect of idebenone after longer term administration onneurological symptoms and signs as assessed by the InternationalCooperative Ataxia…
The primary objective is to determine the effect of a 8-week inspiratory muscle training program on respiratory muscle function in nemaline myopathy patients. The secondary objective is to determine respiratory muscle function in nemaline myopathy…
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,…
To develop an innovative magnetic resonance imaging (MRI) protocol(M-ILD) for accurate phenotyping and sensitive monitoring of therapy in ILD patients.