MANATEE: GYM329 (RO7204239) in Combination with Risdiplam▼ Treatment in Ambulant Children with Spinal Muscular Atrophy
MANATEE (NCT05115110) is a multicenter, two-part, randomized, placebo-controlled, double-blind study that will investigate the effect of GYM329 in combination with risdiplam▼ in treatment-naïve and non-treatment-naïve ambulant patients, aged 2–10 years at screening. Part 1 will assess safety, tolerability and pharmacokinetics/pharmacodynamics of different GYM329 doses in combination with risdiplam▼. Part 2 will assess efficacy and safety of the Part 1-selected dose of GYM329 in combination with risdiplam▼. This poster presentation will provide information about safety and efficacy of GYM329 in combination with risdiplam▼ treatment in ambulant pediatric patients with SMA.
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