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However, in glutathione synthetase deficiency, because the activity of gamma-glutamyl-cysteine synthetase is regulated via feedback inhibition by glutathione, gamma-glutamyl-cysteine and then L-5-oxoproline are overproduced, resulting in the metabolic acidosis and urinary excretion of this compound at levels several hundred-fold above normal (71

Dose increases typically occur after 48 weeks based on injection site tolerance and biomarker response, guided by provider assessment

Anaphylaxis is life-threatening and some people, particularly children, may delay or avoid treatment due to fear of injections, Kelly Stone, associate director of the division of pulmonology, allergy and critical care in the FDAs Center for Drug Evaluation and Research, said in a statement