Steroid-resistant primary focal segmental glomerulosclerosis

The primary objectives of this trial are as follows: - to compare the achievement of a partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio) in patients treated with fresolimumab versus placebo - to compare the safety profile of patients treated with fresolimumab versus placebo The secondary objectives are as follows: - To compare the reduction in proteinuria in patients treated with fresolimumab versus placebo - To evaluate fresolimumab dose-dependent reduction in proteinuria - To compare the change in renal function (estimated glomerular filtration rate [eGFR]) in patients treated with fresolimumab versus placebo - To evaluate the multiple-dose pharmacokinetics of fresolimumab

In 2 brothers with myopathic partial CoQ10 deficiency (39% and 35% of normal restricted to skeletal muscle mitochondria), characterized by proximal and truncal muscle weakness, elevated creatine kinase, lactic acidosis, and myoglobinuria, Di Giovanni et al. (2001) reported a dramatic clinical and pathologic response to CoQ10 (ubidecarenone) supplementation. Before therapy, muscle biopsies of the patients showed fiber hypotrophy, marked lipid accumulation, ragged-red fibers, and myofibers with multiple features of apoptosis. After 8 months of treatment, excessive lipid storage resolved, CoQ10 levels normalized, mitochondrial enzyme levels increased, and the proportion of apoptotic fibers decreased. Di Giovanni et al. (2001) concluded that a depletion of CoQ10 in tissues results in impairment of oxidative phosphorylation and ATP production, an increase in the levels of damaging reactive oxygen species, and a decrease in the inhibition of apoptosis.

Steroid-resistant primary focal segmental glomerulosclerosis

steroid-resistant primary focal segmental glomerulosclerosis

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steroid-resistant primary focal segmental glomerulosclerosissteroid-resistant primary focal segmental glomerulosclerosissteroid-resistant primary focal segmental glomerulosclerosissteroid-resistant primary focal segmental glomerulosclerosissteroid-resistant primary focal segmental glomerulosclerosis

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