3 results
Approved WMOCompleted
Primary objectives of this study:1. Evaluate the efficacy after 26 weeks of Kuvan® treatment + Phe-restricted diet therapy in increasing dietary Phe tolerance, as compared to dietary therapy alone in <4 year-old infants and children with…
Approved WMOCompleted
Explore efficacy, safety, and feasibility (of an eventual future phase 3 trial) of intravenous immunoglobulins as treatment for patients with idiopathic inflammatory myopathies.
Approved WMORecruiting
1. Assessing the efficacy of IVIg in autoimmune epilepsy, both clinically and serologically.2. Identifying an objective marker of therapy response in epilepsy, measuring cortical excitability by TMS-EEG/EMG, in vivo.3. Providing evidence that…