The chronic use of a TGF-β-targeting phosphorodiamidate morpholino oligomer (PMO) and dystrophin exon-skipping PMO reduced muscle fibrosis and loss, restored dystrophin expression in skeletal and ...

Shared pathogenic mechanisms between primary biliary cholangitis (PBC) and primary Sjogren syndrome could allow the development of treatment strategies that may benefit patients with both conditions.

The average telomere loss in patients with SSc was approximately 223 base pairs per year, compared with 30-60 pairs per year in normal aging. Shortened peripheral blood leukocyte telomere length ...

RN3271 showed deep and durable effects in reducing TTR protein with a favorable safety profile. MINNEAPOLIS— RN3271, a novel GalNAc-conjugated siRNA designed to silence transthyretin (TTR) expression ...

After switching to sirolimus or azathioprine the patients showed no evidence of active CIDP, even though they did not receive any other treatment. The use of tacrolimus to avoid rejection following ...

Given the results, the investigation of intranasal carbetocin in PWS has been halted. Intranasal carbetocin did not demonstrate clinical benefit for hyperphagia in patients with Prader-Willi syndrome ...

Subgroup analyses showed this PFS advantage was consistent across patient subgroups and alpha‐fetoprotein levels. For patients with advanced hepatocellular carcinoma (HCC) and a high CRAFITY score, ...

Rituximab was linked to nearly triple the risk of hypogammaglobulinemia in the discovery cohort and a nearly 5-fold higher risk in the larger validation cohort. Patients with multiple sclerosis (MS) ...

This regimen led to a clinically meaningful 1.94-point reduction in Myasthenia Gravis Activities of Daily Living scores over 24 weeks in a simulated phase 3 population. A maintenance dose of 15 ...