Neurology
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
Scientific Publications & Presentations
Built on decades of scientific leadership, Ionis continues to advance the understanding of disease through groundbreaking research and innovation. Explore select publications across our therapeutic areas and technologies to better understand the science behind our medicines and pipeline.
Showing 49-55 out of 55 results
Cardiometabolic Disease
N-acetyl galactosamine-conjugated antisense drug to APOC3 mRNA, triglycerides and atherogenic lipoprotein levels
Cardiometabolic Disease
Characterizing familial chylomicronemia syndrome: Baseline data of the APPROACH study
Neurology
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Neurology
Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease
Neurology
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Neurology
Towards a therapy for Angelman syndrome by targeting a long non-coding RNA