Shuting Xia

TL;DR: Analysis of autopsy tissue from patients exposed to nusinersen showed drug uptake into motor neurons throughout the spinal cord and neurons and other cell types in the brainstem and other brain regions, exposure at therapeutic concentrations, and increased SMN2 mRNA exon 7 inclusion and SMN protein concentrations in the spinal Cord.

TL;DR: A phase 1/2a first-in-man trial and a phase 2 trial aimed to assess the efficacy, safety, and tolerability of two unique antisense oligonucleotides designed to lower Lp(a) concentrations.

TL;DR: APO(a)-LRx reduced lipop protein(a) levels in a dose-dependent manner in patients who had elevated lipoprotein( a) levels and established cardiovascular disease.

TL;DR: Nusinersen treatment over ∼3 years resulted in motor function improvements and disease activity stabilization not observed in natural history cohorts, document the long-term benefit of nusineren in later-onset SMA, including SMA type III.

TL;DR: Lumbar puncture–related adverse event frequency was similar to that previously reported in children, and adverse events were more frequent in older children, children with type 3 spinal muscular atrophy, and with a 21- or 22-gauge needle compared to a 24-g Gauge needle or smaller.