[P2] Poster Presentation
metabolic disease 2

[P-008] Two-year treatment of early infantile Niemann-Pick disease C with miglustat and intrathecal HPBCD

^○Takafumi Sakakibara¹, Noriko Tomomatsu¹, Kenichi Ogiwara¹, Muneaki Matsuo², Aya Narita³, Kousaku Ohno⁴, Midori Shima¹ (1.Department of Pediatrics, Nara Medical University, Nara, Japan, 2.Department of Pediatrics, Faculty of Medicine, Saga University, Saga, Japan, 3.Division of Child Neurology Institute of Neurological Science Tottori University Faculty of Medicine, Yonago, Japan, 4.Japan Organization of Occupational Health and Safety, Sanin Roosai Hospital, Yonago, Japan)