Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study

Author:

Narita Aya1ORCID,Shirai Kentarou1,Itamura Shinji1,Matsuda Atsue1,Ishihara Akiko2,Matsushita Kumi2,Fukuda Chisako3,Kubota Norika4,Takayama Rumiko5,Shigematsu Hideo5,Hayashi Anri6,Kumada Tomohiro6,Yuge Kotaro7,Watanabe Yoriko7,Kosugi Saori8,Nishida Hiroshi8,Kimura Yukiko8,Endo Yusuke9,Higaki Katsumi10,Nanba Eiji10,Nishimura Yoko1,Tamasaki Akiko1,Togawa Masami1,Saito Yoshiaki1,Maegaki Yoshihiro1,Ohno Kousaku1,Suzuki Yoshiyuki11

Affiliation:

1. Division of Child Neurology Institute of Neurological Science Tottori University Faculty of Medicine Yonago Japan

2. Rehabilitation Division Tottori University Hospital Yonago Japan

3. Department of Pathobiological Science and Technology Faculty of Medicine Tottori University Yonago Japan

4. Department of Pediatrics National Hospital Organization Matsue Medical Center Shimane Japan

5. Department of Pediatrics National Epilepsy Center Shizuoka Institute of Epilepsy and Neurological Disorders Shizuoka Japan

6. Department of Pediatrics Shiga Medical Center for Children Moriyama Japan

7. Department of Pediatrics and Child Health Kurume University School of Medicine Kurume Japan

8. Pharmacokinetics and Bioanalysis Center Shin Nippon Biomedical Laboratories, Ltd Kainan Japan

9. Center for Promoting Next‐Generation Highly Advanced Medicine Tottori University Hospital Yonago Japan

10. Division of Functional Genomics, Research Center for Bioscience and Technology Tottori University Yonago Japan

11. Tokyo Metropolitan Institute of Medical Science Tokyo Japan

Funder

Ministry of Health, Labor and Welfare of Japan

Publisher

Wiley

Subject

Clinical Neurology,General Neuroscience

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