Exploring the efficacy and safety of Ambroxol in Gaucher disease: an overview of clinical studies
Gaucher disease (GD) is mainly caused by glucocerebrosidase (GCase) enzyme deficiency due to genetic variations in the GBA1 gene leading to the toxic accumulation of sphingolipids in various organs, which causes symptoms such as anemia, thrombocytopenia, hepatosplenomegaly, and neurological manifest...
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Frontiers Media S.A.,
2024-02-01T00:00:00Z.
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