Neuroscience
WMS Poster 2018: A study of risdiplam (RG7916) in babies with pre-symptomatic spinal muscular atrophy
Oct 03, 2018
RAINBOWFISH is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, pharmacokinetics and pharmacodynamics of risdiplam (RG7916) in asymptomatic infants, aged from birth to 6 weeks of age (at first dose) with genetically diagnosed SMA. RAINBOWFISH will provide data on the early administration of RG7916 alongside the ongoing FIREFISH, SUNFISH and JEWELFISH studies.
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WMS Poster 2018: Risdiplam (RG7916) increases SMN protein levels and improves motor function in babies with Type 1 SMA
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WMS Poster 2018: A Phase 1b/2 study of the anti-myostatin adnectin RG6206 (BMS-986089) in ambulatory boys with Duchenne muscular dystrophy
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WMS Poster 2018: JEWELFISH: risdiplam (RG7916) increases SMN protein in patients with SMA that have previously received therapies targeting SMN2 splicing