Consensus on the use of intrathecal gene therapy for spinal muscular atrophy. (PubMed, Neuropediatrics) - Oct 14, 2026 - "A structured, multimodal monitoring approach-including dorsal root ganglia surveillance, multidisciplinary long-term follow-up, and coordinated transition from pediatric to adult care-is essential for the safe implementation of intrathecal Onasemnogene abeparvovec in SMA."
Journal • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Pediatrics • Rare Diseases
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Andreas Hahn; Andreas Ziegler; Astrid Blaschek; Astrid Pechmann; Claudia Weiß; Cornelia Köhler; Georg M M Stettner; Günther Bernert; Janbernd Kirschner; Jessika Johannsen; Katharina Vill; Maggie Christine Walter; Maja von der Hagen; Marina Flotats Bastardas; Markus Weiler; Matthias Baumann; Susanne Petri; Tim Hagenacker; Ulrike Schara-Schmidt
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