4 results
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…
The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…
The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.
To establish the safety and effectiveness of the Edwards SAPIEN 3/ SAPIEN 3 Ultra Transcatheter Heart Valve (THV) in subjects with moderate, calcific aortic stenosis (AS)