[4P-0452] Evaluation of the efficacy and safety of a new drug candidate for Niemann-Pick disease type C

〇Minami Soga¹, Yoichi Ishitsuka², Mikoto Hamasaki¹, Kaori Yoneda³, Hirokazu Furuya⁴, Muneaki Matsuo⁵, Hironobu Ihn⁶, Noemi Fusaki⁷, Kimitoshi Nakamura³, Naomi Nakagata⁸, Fumio Endo³, Tetsumi Irie², Takumi Era¹ (1.Department of Cell Modulation, Institute of Molecular Embryology and Genetics, Kumamoto University, 2.Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University, 3.Department of Pediatrics, Graduate School of Medical Sciences, Kumamoto University, 4.Department of Cardiology, Neurology and Aging Science, Kochi University Medical School, 5.Department of Pediatrics, Saga University, Faculty of Medicine, 6.Department of Dermatology and Plastic Surgery, Faculty of Life Sciences, Kumamoto University, 7.Keio University School of Medicine, 8.Division of Reproductive Engineering, Center for Animal Resources and Development, Kumamoto University)

Induced pluripotent stem cells, lysosomal storage disease