2 results
Approved WMOCompleted
1. Overall survival (primary efficacy endpoint)2a. Efficacy: progression-free survival (PFS), overall response rate (ORR = CR + PR), and a clincal benefit rate (CBR) will be evaluated as CR + PR + SD for at least 3 months.2b. Safety: adverse events…
Approved WMORecruiting
The aim of this study is to determine whether MI-E leads to a lower number of RTIs as compared to AS combined with MAC in DMD patients with a PECF< 270 l/min over a period of two years.