3 results
Approved WMOCompleted
Primary:To assess the safety and tolerability of Firdapse after single and multiple doses Secondary:Part 1 only: To determine the dose-related PK profile of single, escalating doses of Firdapse;Part 2 only: To assess the steady-state plasma PK and…
Approved WMORecruiting
This study has two major goals. The first goal of this study is to evaluate whether the disabilitating effects of crowding can be reduced by the use of a magnifier early in life. The second goal is to investigate motor and visual development of…
Approved WMORecruiting
The aim of this study is to investigate the efficacy and tolerability of pyridostigmine monotherapy and amifampridine as add-on compared to placebo in patients with MG.