4 results
The study objectives are:- to characterize the biotransformation pathways, the routes and rates of excretion, and total recovery of the 14C-labeled research medication and its radiolabeled metabolites- to characterize the pharmacokinetics (PK) of…
• To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP caused by RPE65 or LRAT gene mutations.• To evaluate duration of visual function improvement (if observed) in subjects with LCA or RP…
The primary objective of this study is:- To evaluate the long-term safety of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA)The secondary objectives of this study are:-To compare the proportion of subjects with wAIHA…
The purpose of this study is to measure clinical efficacy on muscle strength and function, safety, and tolerability of NMD670 compared with placebo in ambulatory participants with Type 3 SMA