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In this retrospective cohort study, we profiled circulating cell-free messenger RNA (ccfmRNA) in serum samples from 17 adults with loSMA type 2 (n = 14) and type 3 (n = 3) and healthy controls (n = 12) using the NanoString neuroimmunology panel before (T0) and after 8\u201310 (T1) and 12\u201314 (T2) months of risdiplam treatment. A separate exploratory subgroup of patients switching from nusinersen to risdiplam (n = 7) was analysed at comparable time points. Differential ccfmRNA expression, gene ontology and network analyses were performed. At baseline, adult loSMA showed a distinct inflammatory and stress-related ccfmRNA signature and reduced expression of selected glial- and vascular-associated transcripts compared to controls. Risdiplam partially normalized immune and proteostasis transcripts, whereas glial and vascular modules remained persistently dysregulated. Exploratory subgroup analyses suggested larger transcriptional deviations in type 2 than in type 3, although these findings are limited by small sample size. In switcher patients, additional transcriptional changes involving epigenetic- and lipid-associated pathways were observed and should be regarded as hypothesis-generating. Serum ccfmRNA profiles distinguish adult loSMA from controls and capture risdiplam-associated molecular responses. These findings support the concept that serum ccfmRNA profiling may provide a useful exploratory framework for studying disease-associated and treatment-associated molecular changes in adult loSMA.<\/jats:p>","DOI":"10.1093\/braincomms\/fcag352","type":"journal-article","created":{"date-parts":[[2026,9,14]],"date-time":"2026-09-14T11:19:18Z","timestamp":1789384758000},"source":"Crossref","is-referenced-by-count":0,"title":["Serum circulating cell-free messenger RNA profile response to risdiplam treatment in adult patients with late-onset spinal muscular atrophy"],"prefix":"10.1093","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3449-9829","authenticated-orcid":false,"given":"Markus","family":"Leo","sequence":"first","affiliation":[{"name":"Department of Neurology, Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Medicine Essen , Essen 45147 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