ReviewFrontiers in molecular medicine2026
Spinal muscular atrophy in the disease-modifying therapy era: successes, limitations and future directions.
Review in Frontiers in molecular medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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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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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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Authors and funding
3 authors.
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Abstract
Spinal Muscular Atrophy (SMA) is a rare and debilitating neurodegenerative disease characterized by the progressive loss of motor neurons in the spinal cord, leading to muscle weakness, respiratory failure, and premature mortality. The pathogenesis of SMA is highly complex and the investigation of downstream pathways and specific cellular mechanisms is still ongoing. In recent years, three FDA-approved disease-modifying therapies, nusinersen, risdiplam, and onasemnogene abeparvovec, have improved the quality of life for patients with SMA and have eased the management of associated symptoms. However, unmet needs remain as comorbidities become increasingly apparent in the era of disease-modifying therapies. Despite the remarkable progress achieved over the past decade, continued research is essential to further improve the quality of life, clinical outcomes, and standard of care for individuals living with SMA.
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