University of Salford

About: University of Salford is a education organization based out in Salford, Manchester, United Kingdom. It is known for research contribution in the topics: Population & Context (language use). The organization has 13049 authors who have published 22957 publications receiving 537330 citations. The organization is also known as: University of Salford Manchester & The University of Salford Manchester.

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

Abstract: To assess the efficacy and safety of enzyme replacement therapy (ERT) with BMN 110 (elosulfase alfa) in patients with Morquio A syndrome (mucopolysaccharidosis IVA). Patients with Morquio A aged ≥5 years (N = 176) were randomised (1:1:1) to receive elosulfase alfa 2.0 mg/kg/every other week (qow), elosulfase alfa 2.0 mg/kg/week (weekly) or placebo for 24 weeks in this phase 3, double-blind, randomised study. The primary efficacy measure was 6-min walk test (6MWT) distance. Secondary efficacy measures were 3-min stair climb test (3MSCT) followed by change in urine keratan sulfate (KS). Various exploratory measures included respiratory function tests. Patient safety was also evaluated. At week 24, the estimated mean effect on the 6MWT versus placebo was 22.5 m (95 % CI 4.0, 40.9; P = 0.017) for weekly and 0.5 m (95 % CI −17.8, 18.9; P = 0.954) for qow. The estimated mean effect on 3MSCT was 1.1 stairs/min (95 % CI −2.1, 4.4; P = 0.494) for weekly and −0.5 stairs/min (95 % CI −3.7, 2.8; P = 0.778) for qow. Normalised urine KS was reduced at 24 weeks in both regimens. In the weekly dose group, 22.4 % of patients had adverse events leading to an infusion interruption/discontinuation requiring medical intervention (only 1.3 % of all infusions in this group) over 6 months. No adverse events led to permanent treatment discontinuation. Elosulfase alfa improved endurance as measured by the 6MWT in the weekly but not qow dose group, did not improve endurance on the 3MSCT, reduced urine KS, and had an acceptable safety profile.

Computer simulation of random packing of unequal particles.

Abstract: A Monte Carlo simulation model for the random packing of unequal spherical particles is presented in this paper. With this model, the particle radii obeying a given distribution are generated and randomly placed within a cubic packing domain with high packing density and many overlaps. Then a relaxation iteration is applied to reduce or eliminate the overlaps, while the packing space is gradually expanded. The simulation is completed once the mean overlap value falls below a preset value. To simulate the random close packing, a ``vibration'' process is applied after the relaxation iteration. For log-normal distributed particles, the effect of particle size standard deviation, and for bidisperse particles, the effects of particle size ratio and the volume fraction of large particles on packing density and on coordination number are investigated. Simulation results show good agreement with that obtained by experiments and by other simulations. The randomness, homogeneity, and isotropy, which have not been evaluated before for packing of distributed particles, are also examined using statistical measures.