SynthesisEuropean journal of pediatrics2025
Combination therapies in spinal muscular atrophy: a systematic review.
Synthesis in European journal of pediatrics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
4 citing papers in PubMed.
- Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment.RNA biology · 2026Review
- 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
- Therapeutic advances in spinal muscular atrophy: a review of clinical, safety, and economic considerations.Frontiers in pharmacology · 2026Review
- A therapeutic antisense oligonucleotide encompassing 2'-NAR molecular medicine · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The purpose of this study was to evaluate the safety, efficacy, and clinical application of combination therapies involving nusinersen, onasemnogene abeparvovec-xioi, and risdiplam in patients with spinal muscular atrophy (SMA), and to assess their potential advantages over monotherapy. This systematic review included studies up to May 2025 from PubMed, Scopus, Web of Science, and ClinicalTrials.gov. Studies involving dual- or triple-combination therapies, either as switching or add-on strategies, were identified and categorized. Data extraction included patient demographics, treatment regimens, motor function outcomes, and adverse events. Study quality was assessed using the Joanna Briggs Institute Critical Appraisal tools. Out of 985 records, 19 studies and 6 ongoing clinical trials met inclusion criteria. A total of 29 individual patients receiving combination therapies (dual: n = 26, triple: n = 3) were analyzed. Switching therapies were the most common, particularly from nusinersen or risdiplam to onasemnogene abeparvovec-xioi. Combination regimens were generally well-tolerated with no consistent evidence of additive toxicity. However, long-term efficacy remains uncertain. Some patients demonstrated improved motor milestones and respiratory function, particularly with early intervention.
conclusionCombination therapies for SMA are emerging as a feasible and generally safe strategy, especially in patients with suboptimal response to monotherapy. While current evidence is encouraging, robust long-term trials are essential to determine their true efficacy, optimal sequencing, and broader impacts, including cost-effectiveness and systemic benefits. WHAT IS KNOWN: •Three disease-modifying therapies are approved for SMA, but monotherapy may not halt disease progression in all patients. WHAT IS NEW: •This systematic review evaluates existing evidence on combination therapies (add-on/switching) for SMA. •Combination therapies appear safe and well-tolerated in short-term studies. •Long-term efficacy and optimal sequencing of dual/triple regimens remain uncertain and warrant further research.
Indexed as
Identifiers
40879810What Socratic holds
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 Socratic graph.