Neurology
Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome
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 37-48 out of 51 results
Neurology
Liver-directed drugs for transthyretin-mediated amyloidosis
Neurology
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Neurology
Eplontersen Data Presented at the XVIII International Symposium on Amyloidosis
Neurology
Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment
Neurology
Angelman syndrome genotypes manifest varying degrees of clinical severity and developmental impairment
Neurology
Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Cardiometabolic Disease
Lipoprotein(a) Reduction in Persons with Cardiovascular Disease
Neurology
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
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