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  1. Extended application of an old medication

Extended application of an old medication

Yoon-Myung Kim 1 Mi-Sun Yum 2 Sun Hee Heo 2 Taeho Kim 2 Hee Kyung Jin 3 Jae-sung Bae 3 Go Hun Seo 2 Arum Oh 2 Hee Mang Yoon, PhD 2 Hyun Taek Lim 2 Hyo-Won Kim, MD, PhD 2 Tatiana Ko 2 Hyeong-Seok Lim 2 Mark Osborn, PhD 4 Jakub Tolar 4 Dr. rer. nat. Claudia Cozma, MD 5 Prof. Arndt Rolfs, MD 5, 6 Ari Zimran, MD 7 Pr. Beom Hee Lee 2 Han-Wook Yoo, MD, PhD 2
1 Gangneung Asan Hospital 2 University of Ulsan 3 Kyungpook National University 4 University of Minnesota Twin Cities 5 CENTOGENE AG 6 University of Rostock 7 The Hebrew University of Jerusalem
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.