Rna

Avidity Biosciences Inc. announced positive data from a phase 1/2 trial, which successfully delivered RNA into muscle for the first time in a study that aimed to treat patients with myotonic dystrophy, the company said in a press release.

Medesis Pharma has received a grant of 300,000 euros from AFM-Téléthon to fund the first phases of preclinical development of its RNA interference treatment for Huntington’s disease, the company announced.

Combined serum circulating micro-RNA served as a novel biomarker for the diagnosis of nonalcoholic steatohepatitis in patients with nonalcoholic fatty liver disease, according to research published in Nature Scientific Reports.

Extending dosing of a novel GalNAc-small interfering RNA brought most patients’ surface antigen levels of hepatitis B virus under control, according to a presenter at the International Liver Congress.

RNA sequencing detected less than half of identifiable genetic mutations found in DNA sequencing of thyroid and brain tumor, thyroid tissue and fine-needle aspiration samples, according to findings published in Thyroid.

Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics.