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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

Results showed that all 9 patients remained relapse-free during a median follow-up of 14.5 months. Ravazulimab appears to be a safe and effective therapeutic alternative for patients with ...

Seventeen of the 22 ultimately diagnosed with cMCD had relatively low basal serum tryptase, and 64% initially tested negative for peripheral blood KIT p.D816V. SM is a rare disorder involving the ...

Elizabeth Hsia, MD, MSCE, speaks at the 2025 International Society of Ultrasound in Obstetrics and Gynecology World Congress (Photo by Brian Murphy PhD) For neonates with HDFN who received postnatal ...

The majority (90%) of neurologists were aware of 40 out of 61 recommendations on the treatment of myasthenia gravis (MG). Most Spanish neurologists know, agree on, and apply guidelines for the ...