SUNFISH Part 2: Efficacy and Safety of Risdiplam (RG7916) in Patients with Type 2 or Non-Ambulant Type 3 Spinal Muscular Atrophy (SMA) (1260)
Eugenio Mercuri, Nina Barišić, Odile Boespflug‐Tanguy, Nicolas Deconinck, Anna Kostera‐Pruszczyk, Riccardo Masson, Elena Mazzone, A. Nascimento, Kayoko Saito, D. Vlodavets, Carole Vuillerot, Sabine Fuerst‐Recktenwald, Sibylle Fuhrer, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Carmen Martin, Wai Yin Yeung, John W. Day
Abstract
To determine the safety and efficacy of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA) treated for 12 months during confirmatory Part 2 of the SUNFISH study.
Topics & Concepts
Spinal muscular atrophySMA*MedicineSpinal cord injuryPhysical therapyInternal medicineSpinal cordPsychiatryCombinatoricsDiseaseMathematicsNeurogenetic and Muscular Disorders ResearchRNA Research and SplicingRNA modifications and cancer