Ambroxol as Therapy for Gaucher Disease-Ambitious but Ambivalent

JAMA Netw Open. 2023 Jun 1;6(6):e2319336. doi: 10.1001/jamanetworkopen.2023.19336.

Authors

Neal J Weinreb¹, Ozlem Goker-Alpan²

Affiliations

¹ Department of Human Genetics, University of Miami Miller School of Medicine, Boca Raton, Florida.
² Lysosomal and Rare Diseases Research and Treatment Center, Fairfax, Virginia.

PMID: 37342045
DOI: 10.1001/jamanetworkopen.2023.19336

No abstract available

Publication types

Comment

MeSH terms

Ambroxol* / therapeutic use
Gaucher Disease* / drug therapy
Glucosylceramidase / genetics
Humans
Mutation

Substances

Ambroxol
Glucosylceramidase