2 results
Approved WMORecruiting
The objective of this pivotal study is to assess the PK and PD as well as safety and tolerability of RP103 compared to Cystagon® in patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support the registration…
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 (…