Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study

Narita, Aya; Shirai, Kentarou; Itamura, Shinji; Matsuda, Atsue; Ishihara, Akiko; Matsushita, Kumi; Fukuda, Chisako; Kubota, Norika; Takayama, Rumiko; Shigematsu, Hideo; Hayashi, Anri; Kumada, Tomohiro; Yuge, Kotaro; Watanabe, Yoriko; Kosugi, Saori; Nishida, Hiroshi; Kimura, Yukiko; Endo, Yusuke; Higaki, Katsumi; Nanba, Eiji; Nishimura, Yoko; Tamasaki, Akiko; Togawa, Masami; Saito, Yoshiaki; Maegaki, Yoshihiro; Ohno, Kousaku; Suzuki, Yoshiyuki

doi:10.1002/acn3.292

Public

Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study

Published in Annals of Clinical and Translational Neurology • Feb 2, 2016

Authors:

Aya Narita

Kentarou Shirai

Shinji Itamura

Abstract

OBJECTIVE: Gaucher disease (GD) is a lysosomal storage disease characterized by a deficiency of glucocerebrosidase. Although enzyme-replacement and substrate-reduction therapies are available, their efficacies in treating the neurological manifestations of GD are negligible. Pharmacological chaperon...

Subject

Medicine

Ambroxol

Glucocerebrosidase

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