Session Details

[O15]一般演題(口演)15 末梢神経・筋③

Fri. Jun 6, 2025 4:40 PM - 5:40 PM JST
Fri. Jun 6, 2025 7:40 AM - 8:40 AM UTC
第2会場
座長:石川 悠加(国立病院機構北海道医療センター神経筋/成育センター小児神経科),井上 美智子(国立病院機構南岡山医療センター小児科・小児神経科)

[O15-01]Diagnosis and CK level trends of cases with hyperCKemia as the main complaint

Naoko Taniguchi, Tomoko Lee, Saeka Yoshitake, Yuki Terakita, Eisuke Terasaki, Sachi Tokunaga, Hideki Shimomura, Yasuhiro Takeshima (Department of Pediatrics, Hyogo College Of Medicine, Hyogo, Japan)

[O15-02]Early administration of mechanical ventilation to 2 patients with spinal muscular atrophy improved their chest deformity

Keisuke Oki, Tomokazu Kimizu, Takuya Horibe, Naoko Yamazaki, Ken Nakajima, Kouji Tominaga, Yukiko Mogami, Keiko Yanagihara (Department of Child Neurology, Osaka Women's and Children's Hospital)

[O15-03]EMBARK Part 2: Safety and efficacy of delandistrogene moxeparvovec in duchenne muscular dystrophy

Hirofumi Komaki1, Jerry R. Mendell2,3, Francesco Muntoni4, Eugenio Mercuri5, Ulrike Schara-Schmidt6, Alex Murphy7, Carol Reid7, Jacob S. Elkins8, Louise R. Rodino-Klapac8 (1.Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan, 2.Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA, 3.The Ohio State University, Columbus, OH, USA, 4.The Dubowitz Neuromuscular Centre, Great Ormond Street Institute of Child Health University College London, London, UK, 5.Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy, 6.Department of Pediatric Neurology, Center for Neuromuscular Disorders in Children and Adolescents, University Clinic Essen, University of Duisburg-Essen, Essen, Germany, 7.Roche Products Ltd, Welwyn Garden City, UK, 8.Sarepta Therapeutics, Inc., Cambridge, MA, USA)

[O15-04]Assessment of clinical course in patients with non-5q spinal muscular atrophy

Ikumi Sako1, Yuko Motohashi1, Kaoru Yamamoto1, Noriko Sumitomo1, Hisako Yamamoto1, Shimpei Baba1, Eri Takeshita1, Takashi Saito1, Hirofumi Komaki1,2 (1.Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan, 2.Tranlational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan)

[O15-05]Long-term safety and tolerability of delandistrogene moxeparvovec in DMD: phase 1 to phase 3 clinical trials

Keiko Ishigaki1, Crystal Proud2, Craig M. McDonald3, Eugenio M. Mercuri4, Francesco Muntoni5, Craig M. Zaidman6, Sachi Dharia7, Stefanie Mason7, Jianfeng Meng7, Alexander P. Murphy8, Emanuel Palatinsky7, Carol Reid8, Christoph Wandel9, Jerry R. Mendell7 (1.Tokyo Women's Medical University, School of Medicine, Department of Pediatrics, Tokyo, Japan, 2.Children's Hospital of The King's Daughters, Norfolk, VA, USA, 3.UC Davis, Davis, CA, USA, 4.Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy, 5.The Dubowitz Neuromuscular Centre, Great Ormond Street Institute of Child Health University College London, London, UK, 6.Washington University in Saint Louis, Saint Louis, MO, USA, 7.Sarepta Therapeutics, Inc., Cambridge, MA, USA, 8.Roche Products Ltd, Welwyn Garden City, UK, 9.F. Hoffmann-La Roche Ltd, Basel, Switzerland)

[O15-06]retrospective analysis of STIR imaging of the lower limb muscles in spinal muscular atrophy.

Yuriko Umeyama1, Yuko Motohashi1, Ikumi Sako1, Takeshita Eri1, Kaoru Yamamoto1, Noriko Sumitomo1, Hisako Yamamoto1, Shimpei Baba1, Takashi Saito1, Noriko Sato2, Hirohumi Komaki1,3 (1.Department of Child Neurology, National Center of Neurology and Psychiatry, Tokyo, Japan, 2.Department of Radiology, National Center of Neurology and Psychiatry, Tokyo, Japan, 3.Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan)