Ambroxol as Therapy for Gaucher Disease-Ambitious but Ambivalent

JAMA Netw Open. 2023 Jun 1;6(6):e2319336. 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