[JO-02] Oral Presentation 02

[JO-012] RAINBOWFISH：Preliminary efficacy and safety in risdiplam treated infants with presymptomatic SMA

Kayoko Saito (1. 東京女子医科大学ゲノム診療科、2. Sydney Children's Hospital Network and UNSW Medicine、 UNSW Sydney、 Sydney、 Australia、3. Russian Children Neuromuscular Center、 Veltischev Clinical Pediatric Research Institute of Pirogov Russian National Research Medical University、 Moscow、 Russia、4. I Motion Hopital Armand Trousseau、 Paris、 France、5. Department of Neurology、 Faculdade de Medicina、 Universidade de Sao Paulo、 Sao Paulo、 Brazil、6. Center for Experimental Neurotherapeutics、 St Jude Children's Research Hospital、 Memphis、 TN、 USA、7. UT Southwestern Medical Center、 Dallas、 TX、 USA、8. Children's Hospital of Fudan University、 Shanghai、 China、9. F. Hoffmann—La Roche Ltd、 Basel、 Switzerland、10. Department of Neurosciences and Neurorehabilitation、 Bambino Gesu Children's Research Hospital IRCCS、 Rome、 Italy)