Neuroscience
AAN Presentation 2019: FIREFISH Part 1: 1-year results on motor function in infants with Type 1 SMA
May 07, 2019
by [Giovanni Baranello, Laurent Servais, John W. Day, Nicolas Deconinck, Eugenio Mercuri, Andrea Klein, Basil Darras, Riccardo Masson, Heidemarie Kletzl, Yumi Cleary, Muna El-Khairi, Timothy Seabrook, Christian Czech, Marianne Gerber, Coralie Nguyen, Kristina Gelblin, Ksenija Gorni and Omar Khwaja]
FIREFISH (NCT02913482) is an ongoing, multicenter, open-label study aiming to assess the safety and efficacy of risdiplam (RG7916) in infants aged 1–7 months with Type 1 SMA and two SMN2 gene copies. This presentation describes one-year motor milestone data in infants from FIREFISH Part 1.
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AAN Presentation 2019: FIREFISH Part 1: 1-year results on motor function in infants with Type 1 SMA