Serum circulating cell-free messenger RNA profile response to risdiplam treatment in adult patients with late-onset spinal muscular atrophy. (PubMed, Brain Commun) - Sep 27, 2026 - "A separate exploratory subgroup of patients switching from nusinersen to risdiplam (n = 7) was analysed at comparable time points. 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."
Journal • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases
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Andreas Roos; Christoph Kleinschnitz; Kai Christine Liebig; Linda-Isabell Schmitt; Markus Leo; Stefanie Hezel; Svenja Neuhoff; Tim Hagenacker; Tobias Ruck
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