4 results
Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40.
In Part 1 of the study, a fixed dose of ABBV-3067 (potentiator) will be co-administered with ABBV-2222 (corrector) in a dose range-finding manner to enable a dose selection for ABBV-2222 for Part 2 and future combination studies. In addition, ABBV-…
Primary• To assess the safety and tolerability in healthy volunteers of nizaracianine administered IV in three divided dosesSecondary• To perform pharmacokinetic analyses of blood and urine• To determine if bolus injection of nizaracianine enables…
The purpose of this first-in-human study is to evaluate the safety, tolerability, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses (MADs) of DYNE-101 administered intravenously (IV) to participants with DM1.…