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Extended application of an old medication

Yoon-Myung Kim Mi-Sun Yum Sun Hee Heo Taeho Kim Hee Kyung Jin Jae-sung Bae Go Hun Seo Arum Oh Hee Mang Yoon, PhD Hyun Taek Lim Hyo-Won Kim, MD, PhD Tatiana Ko Hyeong-Seok Lim Mark Osborn, PhD Jakub Tolar Dr. rer. nat. Claudia Cozma, MD Prof. Arndt Rolfs, MD Ari Zimran, MD Pr. Beom Hee Lee Han-Wook Yoo, MD, PhD
October 24, 2019

Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy

While ambroxol has been in medical use for respiratory diseases for over 40 years, its recently understood mode of action suggests a wider applicability. For Gaucher disease (GD), a metabolic disorder, ambroxol has now been shown to improve neurological symptoms, and to reduce the levels of CENTOGENE’s GD-specific biomarker Lyso-Gb1. The study was published in Journal of Medical Genetics.