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. 2016 Feb 2;3(3):200–215. doi: 10.1002/acn3.292

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© 2016 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals, Inc on behalf of American Neurological Association.

This is an open access article under the terms of the Creative Commons Attribution‐NonCommercial‐NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.

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Overview of the dosing regimen for each patient. Ambroxol administration was initiated with a starting dose of 3 mg/kg/day divided into three equal doses in the first three patients (patients 1, 2, and 3), because safety information in humans about long‐term high‐dose ambroxol administration was limited. The dose was subsequently increased in increments of 3 mg/kg to reach the target doses (25 mg/kg/day or a maximum dose of 1300 mg/day) over several months to years. For the remaining two patients, ambroxol was initiated at 9 mg/kg/day in patient 4 or 25 mg/kg/day in patient 5. ERT and concomitant medications were continued during the study.