Concepedia

Publication | Open Access

Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study

DOIFull Paper Access

212

Citations

34

References

2016

Year

Aya Narita, Kentarou Shirai, Shinji Itamura, Atsue Matsuda, Akiko Ishihara, Kumi Matsushita, Chisako Fukuda, Norika Kubota, Rumiko Takayama, Hideo Shigematsu,
Annals of Clinical and Translational Neurology

Abstract

Pharmacological chaperone therapy with high-dose oral ambroxol shows promise in treating neuronopathic GD, necessitating further clinical trials.

References

YearCitations

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