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 ...

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 ...

The consequences of cystic fibrosis (CF) are extensive and affect almost every organ system. The clinical picture is broad and may present with acute or chronic manifestations. Gastrointestinal and ...

Momelotinib treatment leads to clear clinical benefits in patients with myelofibrosis (MF), according to results from a real-world study. Rare Disease Advisor, a trusted source of medical news and ...

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) ...

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 ...

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 ...

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 ...

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 ...