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-…
Objectives of part A:- evaluate the safety, tolerability and pharmacokinetics of 2, 3 or 4 mg AZ-009 after 5 days of dosing in people with Parkinson's disease- evaluate the safety, tolerability and pharmacokinetics of 2, 3 or 4 mg AZ-009 after…
Primary:• To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg over a 48-week treatment period vs. placebo controls in ambulant boys ages 4 to <8 years with DMD using the Time to Stand Test (…