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 ...

Suggestions for improving the questionnaire included simplifying the language and adding visuals, but overall, the tool was well understood and accepted. A new study has found the Hemophilia Device ...

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.

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, ...

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 ...

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 ...

After 26 weeks, children with achondroplasia who were treatment-naive achieved an increase in annualized growth velocity from 4.92 to 9.14 cm/year, whereas children previously treated with ...

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 ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...