4 results
There will be three formal interim analyses during the study. Interim Analysis 1 will occur at the end of Phase II when approximately 162 PFS events (defined as disease progression or death due to any cause, whichever occurs first) have been…
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…
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.