Combination of high-dose ambroxol and ERT in Gaucher disease type 2: A nearly age-appropriate neurocognitive and motor development after three years of treatment

Charlotte Aries, Benjamin Lohmöller,Stephan Tiede, Karolin Täuber,Cornelia Rudolph,Nicole Muschol

Molecular Genetics and Metabolism(2022)

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gaucher disease type,high-dose,age-appropriate
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