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Sponsored AI AM CLAUDE, A Beginner's Guide: Learn the basics of working with AI AI AM CLAUDE, A Beginner's Guide: Learn the basics of working with AI

Author

T. Hagenacker

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Open access Aug 2026

Immune Cell-Specific SMN Expression and Sustained Immune Alterations in Treated Spinal Muscular Atrophy.

Mutations in the survival of motor neuron 1 (SMN1) can reduce functional SMN protein levels, which causes Spinal Muscular Atrophy (SMA), a disease affecting the nervous system and peripheral tissues, including the immune system. Yet, SMN expression across immune cell subsets and the impact of SMN-modulating therapies o...

I. Tapken, Katharina Rahmel-Stein, Christine Ehlers et al. · 0 citations
Open access Sep 2026

Serum circulating cell-free messenger RNA profile response to risdiplam treatment in adult patients with late-onset spinal muscular atrophy

Abstract SMN-enhancing therapies have improved outcomes in adult late-onset spinal muscular atrophy (loSMA), but minimally invasive biomarkers that reflect systemic disease and treatment effects are lacking. In this retrospective cohort study, we profiled circulating cell-free messenger RNA (ccfmRNA) in serum samples f...

M. Leo, Linda-Isabell Schmitt, K. C. Liebig et al. · 0 citations

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