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WMS 2021

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Sep 23 / Roche and Genentech
RAINBOWFISH: A study of risdiplam▼ in infants with presymptomatic spinal muscular atrophy (SMA)
RAINBOWFISH (NCT03779334) is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, PK and PD of risdiplam▼ in infants with genetically diagnosed SMA who are not yet presenting with symptoms. This oral presentation reports baseline demographics of enrolled infants with presymptomatic SMA and reports preliminary efficacy and safety data in presymptomatic infants treated with risdiplam. Bulbar function data from infants treated with risdiplam are presented for the first time.

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Sep 20 / Roche and Genentech
Phase 1/2a trial of SRP-9001 in patients with Duchenne muscular dystrophy: 3-year safety and functional outcomes
rAAVrh74.MHCK7.micro-dystrophin (SRP-9001), an investigational gene transfer therapy, was designed to safely express a functional micro-dystrophin protein in skeletal and cardiac muscle. The objective of the Phase 1/2a clinical trial (NCT03375164) is to evaluate the safety of systemic gene transfer of SRP-9001 in patients with Duchenne muscular dystrophy (DMD). Four patients with DMD (4–7 years old, ambulatory) were enrolled in this single-dose, open-label study. An update on long-term (3-year) functional data from this study will be presented.

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Sep 20 / Roche and Genentech
Pooled safety data from the risdiplam▼ clinical trial development program
FIREFISH (NCT02913482) is assessing the safety, tolerability, PK/PD and efficacy of risdiplam▼ in infants with Type 1 SMA aged 1–7 months. SUNFISH (NCT02908685) is assessing the safety, tolerability, PK/PD and efficacy of risdiplam in patients with Type 2 or 3 SMA aged 2–25 years. JEWELFISH (NCT03032172) is assessing the safety, tolerability and PK/PD of risdiplam in in non-naïve patients with SMA, aged 6 months to 60 years. This poster presents updated pooled safety analyses from FIREFISH Parts 1 and 2, SUNFISH Parts 1 and 2 and JEWELFISH. This analysis will add to the understanding of the longer-term safety profile of risdiplam in individuals with SMA
02:40 PM
Duration 10mins Virtual
RAINBOWFISH: A study of risdiplam▼ in infants with presymptomatic spinal muscular atrophy (SMA)
Servais L, Al-Muhaizea M, Farrar MA, Nelson L, Prufer A, Finkel RS, Wang Y, Zanoteli E, El-Khairi M, Gerber M, Gorni K, Kletzl H, Palfreeman L, Scalco RS, Bertini E on behalf of the RAINBOWFISH Study Group

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