4 results
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…
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…
Primary Objectives*To evaluate the safety and tolerability of multiple oral doses of GLPG1837 in subjects with CF and at least one copy of the S1251N mutation.Secondary Objectives*To assess changes in sweat chloride from baseline (Day 1) as the…
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.