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Spinal Muscular Atrophy

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Mar 16 / Roche and Genentech
Real-world risdiplam▼ effectiveness in adults with spinal muscular atrophy (SMA) from the Pediatric Neuromuscular Clinical Research (PNCR) registry
Adults currently account for ~50% of the SMA population worldwide. This study investigated motor function outcomes in adults with SMA treated with risdiplam▼ in the real world. These analyses used data from individuals enrolled in the PNCR network registry.

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Mar 16 / Roche and Genentech
SUNFISH Parts 1 and 2: 5-year efficacy and safety data of risdiplam▼ in Types 2 and 3 spinal muscular atrophy (SMA)
SUNFISH was a multicenter, two-part, randomized, placebo-controlled, double-blind clinical trial of risdiplam▼ in a broad and heterogeneous population with Types 2 and 3 SMA (aged 2–25 years at enrollment). This is the first presentation of motor function and safety data at the end of the 5-year SUNFISH study. Children, teenagers and adults with SMA showed improvement or stabilization in motor function outcomes, which is in contrast to the decline observed in an untreated population.

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Nov 10 / Roche and Genentech
Risdiplam▼ clinical trial outcomes in individuals with spinal muscular atrophy (SMA) and four SMN2 copies
This poster presents efficacy and safety data from risdiplam▼ clinical trials that explored the impact of risdiplam▼ on outcomes in symptomatic and presymptomatic individuals with SMA and four SMN2 copies.

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Oct 27 / Roche and Genentech
RAINBOWFISH: 2-year efficacy and safety data of risdiplam▼ in infants with pressymptomatic SMA
RAINBOWFISH is a multicenter, open-label, single-arm study, which aims to assess the efficacy and safety of risdiplam▼ in infants with genetically diagnosed, presymptomatic SMA. Here we present RAINBOWFISH results after 2 years of risdiplam▼ treatment, showing that most infants were able to achieve sitting, standing and walking motor milestones.

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Oct 27 / Roche and Genentech
Long-term comparative efficacy and safety of risdiplam▼ versus nusinersen in children with Type 1 SMA
Matching-adjusted indirect comparisons were conducted to compare treatment outcomes between risdiplam▼ and nusinersen in children with Type 1 SMA. Patient-level risdiplam▼ data from 58 children in FIREFISH (Parts 1 and 2; NCT02913482) were compared with published nusinersen data from 81 children in the integrated SHINE (NCT02594124)-ENDEAR (NCT02193074) study. These analyses aimed to compare long-term efficacy and safety of risdiplam▼ versus nusinersen in children with Type 1 SMA after 4 years of treatment.

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Oct 25 / Roche and Genentech
Long-term comparative efficacy and safety of risdiplam▼ versus nusinersen in children with Type 1 SMA
Matching-adjusted indirect comparisons were conducted to compare treatment outcomes between risdiplam▼ and nusinersen in children with Type 1 SMA. Patient-level risdiplam▼ data from 58 children in FIREFISH (Parts 1 and 2; NCT02913482) were compared with published nusinersen data from 81 children in the integrated SHINE (NCT02594124)-ENDEAR (NCT02193074) study. These analyses aimed to compare long-term efficacy and safety of risdiplam▼ versus nusinersen in children with Type 1 SMA after 4 years of treatment.
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