3 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 main objective of the present study is to compare the clinical outcome of bone marrow stimulation alone versus bone marrow stimulation and bone marrow aspirate concentrate (BMAC) for small symptomatic osteochondral defects of the talus.
Main Study Objective• To confirm the dose and assess response to single agent 177Lutetium-DOTATATE treatmentin patients with relapsed or refractory high-risk neuroblastomaSecondary Study Objectives:• To assess long term survival and response• To…