5 results
The objective of the study is to assess the safety and tolerability, and to characterize the pharmacokinetics (PK) and pharmacodynamics (e.g., Cerebrospinal fluid [CSF] levels of IL-1β, TSPO positron emission tomography [PET] imaging) of selnoflast…
The objective of this study is to validate a novel MRS-based method to quantify NAD+ by selectively suppressing the coupled *-ATP spin system, which is overlapping with the NAD+/NADH signals in vivo. For NAD+/NADH quantification to be valuable in…
Study AG348-C-017 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical efficacy and safety of mitapivat in subjects with α- or β NTDT. The primary objective of the study is to compare the…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in pediatric subjects with PK deficiency who are not regularly transfused
Study AG348-C-018 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical efficacy and safety of mitapivat in subjects with α- or β-TDT. The primary objective of the study is to compare the…