Neuroscience

AAN Presentation 2019: FIREFISH Part 1: Survival, ventilation and swallowing ability in infants with Type 1 SMA receiving risdiplam (RG7916)

May 07, 2019 by [Laurent Servais, Giovanni Baranello, 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 data on event-free survival rates and swallowing ability in infants from FIREFISH Part 1.

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