書誌情報

O-234. FIREFISH試験 : 脊髄性筋萎縮症 (SMA) I型小児患者におけるリスジプラム (RG7916) の有効性及び安全性

竹島泰弘1, Servais Laurent2, Baranello Giovanni3, Mazurkiewicz-Beldzinska Maria4, Rose Kristy5, Vlodavets Dmitry6, Xiong Hui7, El-Khairi Muna8, Fuerst-Recktenwald Sabine9, Gerber Marianne9, Gorni Ksenija9, Kletzl Heidemarie9, Scalco Renata9, Darras Basil T10
1兵庫医科大学小児科, 2Reference Center for Neuromuscular Disease, Centre Hospitalier Regional de La Citadelle, 3Carlo Besta Neurological Research Institute Foundation, Developmental Neurology Unit, 4Department of Developmental Neurology, Medical University of Gdansk, 5Paediatric Gait Analysis Service of New South Wales, The Children's Hospital at Westmead Sydney, 6Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute of Pirogov Russian National Research Medical University, 7Department of Pediatrics, Peking University First Hospital, 8Roche Products Ltd., 9F. Hoffmann-La Roche Ltd., 10Boston Children's Hospital, Harvard Medical School
脳と発達 52(suppl): S298-S298, 2020.

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