ArticleMolecular therapy. Advances2026
Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy.
Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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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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Who cites it
1 citing paper in PubMed.
- Peripheral Nerve Injury and Denervation-Induced Sarcopenia with a Focus on Pharmacological Dual-Target Strategies.Biomolecules & therapeutics · 2026Review
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Spinal muscular atrophy (SMA) is a devastating genetic disorder characterized by loss of motor neurons and muscle atrophy. In the most severe form, affected infants experience progressive weakness and, if untreated, typically do not survive beyond 2 years of age. Although several disease-modifying therapies are currently available, treatment response varies and there are no clinically available molecular biomarkers to accurately assess therapeutic efficacy. Extracellular vesicles (EVs) are small, membrane-bound nanoparticles released from all cell types, and contain a diverse cargo reflective of their cell of origin. We have followed a cohort of adults with SMA type 3 over 2 years of treatment with nusinersen. At baseline prior to treatment, individuals with SMA exhibit a trend toward increased concentration of nanoparticles in blood plasma and cerebrospinal fluid relative to healthy controls, and a significant decrease in plasma nanoparticle concentration following treatment. We identified several proteins commonly associated with EVs that were significantly different between individuals with SMA and healthy controls, and 21 EV-associated proteins with significantly altered levels in plasma over the course of nusinersen treatment. These findings suggest that nanoparticles and several EV-associated marker proteins hold promise as potential biomarkers for disease state and treatment response in individuals undergoing nusinersen therapy.
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