ArticleBMC neurology2025
Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series.
Article in BMC neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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.
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Who cites it
6 citing papers in PubMed.
- Ten years of disease-modifying therapy in spinal muscular atrophy: lessons learned and future directions.Nature reviews. Neurology · 2026Review
- Risdiplam Add-On Therapy Following Onasemnogene Abeparvovec in Children With Spinal Muscular Atrophy and 2 SMN2 Copies: A Multi-Center Case Series.Muscle & nerve · 2026Article
- Safety and efficacy of intravenous onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy type 1: interim analysis from LT-001, a long-term follow-up study of patients from the START study.EClinicalMedicine · 2026Article
- Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series.Advances in therapy · 2026Article
- Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registry.Brain : a journal of neurology · 2026Article
- The effectiveness and value of therapies for spinal muscular atrophy.Journal of managed care & specialty pharmacy · 2026Article
Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundSpinal muscular atrophy (SMA) is caused by deletions or mutations in the survival of motor neuron (SMN) 1 gene resulting in progressive motor function loss, and additional disease-related complications, including dysphagia and respiratory failure. With three US FDA-approved disease-modifying therapies (DMTs) available for SMA, patients, caregivers and healthcare providers have become increasingly interested in using a combination of DMTs to maximize clinical benefit. Current data on combination therapy are limited, and additional studies are needed. CASE PRESENTATION: This multicenter, retrospective case series presents real-world outcomes in children with SMA who received onasemnogene abeparvovec (OA; ZOLGENSMA
conclusionsMany children included in this case series had improvements in motor, respiratory and/or bulbar function after adding risdiplam following OA. No new safety concerns were observed. The real-world evidence generated from this case series provides additional information on risdiplam's risk-benefit profile after OA administration in children with SMA. Future studies with a larger cohort should be conducted.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.