Early Initiation of High-dose Oral Ambroxol in Combination with Enzyme Replacement Therapy in a Neuropathic Gaucher Infant
Overview
Affiliations
Higashi K, Sonoda Y, Kaku N, Fujii F, Yamashita F, Lee S Mol Genet Genomic Med. 2024; 12(4):e2427.
PMID: 38553911 PMC: 10980885. DOI: 10.1002/mgg3.2427.
Exploring the efficacy and safety of Ambroxol in Gaucher disease: an overview of clinical studies.
Mohamed F, Al-Jasmi F Front Pharmacol. 2024; 15:1335058.
PMID: 38414738 PMC: 10896849. DOI: 10.3389/fphar.2024.1335058.
The use of Ambroxol for the treatment of Gaucher disease: A systematic review.
Abelleyra Lastoria D, Grewal S, Hughes D EJHaem. 2024; 5(1):206-221.
PMID: 38406552 PMC: 10887350. DOI: 10.1002/jha2.852.
Changing clinical manifestations of Gaucher disease in Taiwan.
Lu W, Chien Y, Tsai F, Hwu W, Chou Y, Chu S Orphanet J Rare Dis. 2023; 18(1):293.
PMID: 37715271 PMC: 10502973. DOI: 10.1186/s13023-023-02895-z.
Gaucher disease - more than just a rare lipid storage disease.
Roh J, Subramanian S, Weinreb N, Kartha R J Mol Med (Berl). 2022; 100(4):499-518.
PMID: 35066608 DOI: 10.1007/s00109-021-02174-z.