3 results
The primary objective of the study is Study to determine the Safety, Tolerability, Pharmacokinetics and Effect on Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD)
Part 1: The primary objective of this study is to evaluate the efficacy of natalizumab extended interval dosing (EID) in subjects who have previously been treated with natalizumab standard interval dosing (SID) for at least 12 months, in relation to…
The three key outcome measurements and challenges of this proof of concept study are to achieve a decrease in ongoing HO bone formation, decrease in numbers of disease *flare-up* episodes and to prevent the continuation of FOP induced contractures.…