5 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…
Primary: Time to first occurrence of MACE (cardiovascular death, myocardial infarction, or stroke) [Non-inferiority].Secondary: Time to first occurrence of MACE [Superiority], MACE or urgent revascularization for unstable angina, individual…
The overall objective is to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and femaleparticipants less than or equal to 6 months of age with IOPD.
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.
Long-term safety and pharmacokinetics (PK) of neoGAA