3 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…
Main objective:Parts 1, 3, 4, 5 en 6: AMG 160 monotherapy• Evaluate the safety and tolerability of AMG 160 in adult subjects• Part 1 only: Determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D)Part 2: AMG 160 in combination…
Objectives for both the Treatment Period and Extension Period:Primary Objectives: - To assess safety and tolerability of the study medicine in patients with later onset (e.g., Type 2 and Type 3) spinal muscular atrophy (SMA)- To assess the efficacy…