2 results
Approved WMORecruiting
The purpose of this Phase II/III study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7204239, a humanized monoclonal antibody that binds to human latent myostatin, in combination with risdiplam (…
Approved WMORecruiting
To evaluate safety, tolerability pharmacokinetic profile; to explore pharmacodynamics; to evaluate major effects of Guanabenz on quantitative MRI and clinical parameters in pediatric patients with VWM; explore biomarkers in blood and cerebrospinal…