For the last 3 years, the PBC Foundation, based in Edinburgh, Scotland, has conducted an annual summit that addresses the challenges and potential remedies for those living with primary biliary ...

The authors remarked that the results called for further education on MG symptoms for physicians outside the neurology setting. Advanced age and increased time between symptom onset appear to ...

Patients with NMOSD demonstrated significantly higher adrenergic dysfunction compared to patients with MS. In addition, the cardiovascular autonomic dysfunction scores of patients with NMOSD were also ...

Coexisting ATM and del(11q) aberrations were more common in unmutated IGVH CLL. These results support a refinement of current prognostic frameworks in CLL by distinguishing the clinical relevance of ...

Life with alpha-1 antitrypsin deficiency AATD) has generally not been easy for me. I have written several columns about trying to communicate complicated things to others (such as family members, ...

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 ...

Extremely low baseline neutrophil count was a strong predictor of early death, underscoring the need for early risk stratification. Treatment with porcine antihuman T-lymphocyte globulin (ATG) and ...

Riliprubart, a first-in-class humanized IgG4-monoclonal antibody, selectively inhibits activated-C1s within the classical complement pathway. Treatment with riliprubart showed improvement in roughly ...

Luke Chen, MD, MMEd, is a clinical hematologist and professor of medicine at Dalhousie University in Nova Scotia, Canada. Rare Disease Advisor correspondent Tori Rodriguez interviews clinical ...

Generalized pustular psoriasis is the most serious form of psoriasis, with more than half of all patients with the condition needing hospitalization. About half of all people with GPP also have plaque ...

The authors observed a cost increase of approximately 5600 USD for each quality-adjusted life year (QALY) gained. Adjunctive cannabidiol (CBD) appears to be a viable and cost-effective alternative to ...

A substantial proportion of patients with CIDP are refractory to first-line treatments. New drugs tried in CIDP are first aimed at refractory patients. The use of complement-targeting therapies, such ...