ArticleAnnals of clinical and translational neurology2026
Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan.
Article in Annals of clinical and translational neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04174157 (A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy), which is not on this map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Who cites it
1 citing paper in PubMed.
- Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan.Annals of clinical and translational neurology · 2026Article
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Authors and funding
9 authors.
Funding
Abstract
objectiveThere are limited real-world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one-time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real-world outcomes for OA in Japan.
methodsWe report interim, 5-year results of Japanese post-marketing surveillance of OA (part of the RESTORE registry: NCT04174157).
resultsEighty patients were registered and treated with OA (monotherapy: 30%; bridge or switch to OA: 54%). The median (min, max) age (months) was 3.0 (0, 18) at symptom onset and 10.0 (0, 24) at OA infusion. Forty patients each (50.0%) had two or three survival motor neuron 2 (SMN2) gene copies. Ten patients were identified by newborn screening. Adverse events related to OA were reported in 98.8% (serious: 26.3%; no deaths). Adverse events of special interest occurred in 92.5%, including hepatotoxicity (90.0%), transient thrombocytopenia (62.5%), cardiac adverse events (33.8%), and thrombotic microangiopathy (5.0%). Event-free survival at 3 years since OA administration was 93.0%. There was one death from disease progression. Of 39 patients with two or more developmental milestones, 64.1% achieved new developmental milestones and 15.4% maintained their milestones. Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders scores increased by ≥ 4 points in 81.8% (54/66).
interpretationThe safety profile of OA in Japanese patients with SMA mirrored that of earlier studies. In our real-world observations, patients showed gains in or maintenance of motor milestones or motor function scores that were sustained over the observation period.
trial registrationNCT04174157 (ClinicalTrials.gov).
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