Antisense Oligonucleotides

Although inhaled antisense oligonucleotide treatment is safe, its efficacy needs to be determined in patients with the cystic fibrosis splicing mutation 3849+10kb C-to-T, according to a press release from the Cystic Fibrosis Foundation.

Wave Life Sciences has announced positive results from its phase 1b/2a clinical trial of WVE-003, a potential disease-modifying therapeutic for Huntington’s disease.

Vupanorsen (Ionis) for reduction of non-HDL in patients with hyperlipidemia already on statin therapy.

WASHINGTON — Treatment with vupanorsen, a novel antisense oligonucleotide targeting hepatic ANGPTL3, achieved modest reductions in non-HDL at 24 weeks at all doses studied, according to results of the TRANSLATE-TIMI 70 trial.

Approximately 16 of every 100,000 children are diagnosed with Duchenne muscular dystrophy. Diagnosis typically occurs in boys aged 2 to 3 years.

Dilated perivascular spaces correlated with disease severity in Huntington’s disease and could affect the administration of certain treatments, according to findings published in Neurology.