3 results
Approved WMOCompleted
The purpose of the study is to assess the efficacy and safety in adolescents of AFQ056 in treating behavioral symptoms characteristic of patients with FXS. Data from this study will be used to support the registration of AFQ056 in this population…
Approved WMOWill not start
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
Approved WMOCompleted
Primary Objective- To evaluate the feasibility and test-retest variability of nerve excitability threshold tracking - To investigate the sensitivity of nerve excitability measures to detect the effects of mexiletine- To investigate the sensitivity…