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Enrique Caballero, an endocrinologist at Harvard-affiliated Brigham and Women's Hospital

Some patients tolerate injectable versions better if they experienced digestive side effects with oral tablets

When Aqualyx is injected into the fatty tissue, the deoxycholic acid acts as a detergent, dissolving the cell wall and causing the cell to burst

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This enzyme is a zinc-dependent metallopeptidase that is expressed in many different tissues, with especially high expression level in the kidney

Todays approval represents a significant step forward in enabling children to be treated with Brineura as early as possible, when we can have the greatest impact in altering the natural course of disease, Hank Fuchs, president of worldwide research and development at BioMarin, said in a statement