4 results
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 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…
To analyse in what proportion of NMOSD patients with an episode of acute inflammation circulating pathogenic anti-AQP4 IgG antibodies are depleted below detection limits, as measured with a state-of-the-arts cell-based assay, in the timeframe within…
Primary objective• To determine the 1-year graft failure-free survival in highly sensitised kidney transplant patients, pre-treated with imlifidase to turn a positive crossmatch against a deceased donor negativeSecondary objectives relating to…