Events 07/03/23-07/31/23 Practical Research Project for Rare/Intractable Diseases Fiscal 2022 Outcomes Conference

Session 1
Dr. TANIGUCHI Masahiko（Hospital Director, St. Mary's Hospital）
Presentation Title：「Creation of evidence on Okinawa-type neurogenic muscular atrophy by utilizing the patient registry」

Dr. INOUE Haruhisa（Professor, Center for iPS Cell Research and Application, Kyoto University）
Presentation Title：「HMSN-P Registry Study toward Trial-Ready Cohort Construction」

Session 2
Dr. OGI Tomoo（Professor, Department of Genetics, Research Institute of Environmental Medicine, Nagoya University）
Presentation Title：「Understanding of the pathogenesis of rare intractable diseases caused by genome instability」

Session 3
Dr. KAJI Ryuji（Designated Professor, Department of Neurodegenerative Disorders Research, Graduate School of Biomedical Sciences, Tokushima University）
Presentation Title：「Clinical development of ultra-high dose methylcobalamin in ALS」

Session 4
Dr. YOKOTA Takanori（Professor, Department of Neurology and Neurological Science, Tokyo Medical and Dental University）
Presentation Title：「Developing antisense oligonucleotide therapy against spinocerebellar ataxia type31」

Session 6
Dr. MIZOGUCHI Akira（Department of Personalized Cancer Immunotherapy, Graduate School of Medicine, Mie University）
Presentation Title：「Development of a novel cellular imaging technology realizing more accurate surgical operations on the Hirschsprung disease, a rare intractable infantile disease ----- A technology supporting real-time pathological diagnosis using curcumin vital staining and multiphoton laser scanning endomicroscopy -----」

Closing Remarks