4 results
Primary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood phenylalanine concentrations.Secondary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood tyrosine…
Currently, caplacizumab is being developed for treatment of acquired thrombotic thrombocytopenic purpura (TTP). TTP is a rare and potentially life-threatening thrombotic microangiopathy, in which accumulation of ULvWF multimers leads to an increased…
This is a follow-up (FU) study for subjects who completed Study ALX0681-C301 (HERCULES) according to the protocol (i.e., completed the Final [28 day] FU visit). Subjects who completed Study ALX0681-C301 will be given the option to participate in…
• Randomized Placebo-Controlled Period:Primary:- to demonstrate the efficacy of mavorixafor in patients with Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) syndrome as assessed by increasing levels of circulating neutrophils…