Early initiation of ambroxol treatment diminishes neurological manifestations of type 3 Gaucher disease: A long-term outcome of two siblings
Danijela Petković Ramadža, Marija Zekušić, Tamara Žigman, Ana Škaričić, Ana Bogdanić, Gordana Mustać, Katarina Bošnjak-Nađ, David Ozretić, Kousaku Ohno, Ksenija Fumić, Ivo Barić
Topics & Concepts
AmbroxolEnzyme replacement therapyMedicineDiseasePediatricsSiblingGlucocerebrosidaseInternal medicineAnesthesiaPsychologyDevelopmental psychologyLysosomal Storage Disorders ResearchStudies on Chitinases and ChitosanasesCellular transport and secretion