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AVXS-101 and nusinersen for spinal muscular atrophy type 1

EAN 2019
For spinal muscular atrophy (SMA), nusinersen is currently the only registered therapy. Another very promising therapy in late-stage development is onasemnogene abeparvovec (AVXS-101) gene-replacement therapy. AVXS-101 for non-ambulatory patients with SMA was feasible, well tolerated, and might improve motor functions, as concluded from interim data analysis of the phase 1/2a STRONG study. In this open-label study, SMA patients of ≥6 to <60 months of age who could not stand or walk, received one of 3 doses intrathecal AVXS-101 (dose A: 6.0x1013; B: 1.2x1014; C: 2.4x1014 vector genomes [vg]). As of 24 March 2019, 31 patients from 11 sites were enrolled. All participants experienced treatment-emergent adverse events (AEs) but none were fatal. In 4 patients, 7 serious treatment-emergent AEs occurred, which were all resolved. Patients aged ≥24 ...

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