SynthesisJournal of neuromuscular diseases2025
Systematic literature review of the impact of spinal muscular atrophy therapies on bulbar function.
Synthesis in Journal of neuromuscular diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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.
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Authors and funding
5 authors.
Funding
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Abstract
backgroundImprovement and maintenance of bulbar function are important goals of disease-modifying treatments (DMTs) for spinal muscular atrophy (SMA), but standardized and validated measures for assessing bulbar function do not exist, nor does a widely accepted definition of bulbar function in SMA. As such, the impact of DMTs on bulbar function has not yet been comprehensively evaluated.
objectiveWe conducted a systematic literature review (SLR) to identify evidence about the impact of DMTs for SMA on bulbar function.
methodsWe used the Preferred Reporting Items for Systematic Reviews and Meta-Analysis guidelines to conduct this review. Embase
resultsWe identified 51 studies (across 83 publications) that evaluated SMA DMTs and bulbar function for more than 1600 patients. The ability to feed orally, the ability to tolerate liquids, and the need for nutrition support were commonly reported. Most infants treated with any DMT before SMA symptom onset preserved bulbar function. Infants, children, and adults treated after SMA symptom onset experienced variable results in terms of bulbar function outcomes.
conclusionsThe definition and assessment of bulbar function are not standardized. Therefore, the tools, scales, methods, and timing used for bulbar function assessments varied among studies. Larger prospective studies using standardized and age-based assessments with longer follow-up periods are needed to assess the clinical stability of bulbar function for patients with SMA who receive DMTs.
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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.