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 ...

Testing for AAV is recommended in patients with hepatitis B even in the absence of liver symptoms. A patient with chronic hepatitis B infection (HBV) initially presented with antineutrophil ...

New research indicates that obinutuzumab is a potentially effective and cost-effective treatment for patients with rituximab-resistant AAV. Obinutuzumab therapy demonstrated immunological superiority ...

Mereo BioPharma has presented data from the phase 2 “ASTRAEUS” trial of alvelestat in alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD), as well as post hoc analyses demonstrating the ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

Patients with early-stage PBC and ductopenia had reduced biochemical response to ursodeoxycholic acid treatment. Ductopenia is a significant risk factor for worse biochemical profiles and poor ...

A patient presented with task-related isolated transient weakness in her right hand, which started 15 years before and lasted for 9 years without worsening or improvement. Distal myasthenia gravis (MG ...

Larry Luxner, senior correspondent for Rare Disease Advisor, talks with Claire Booth, MBBS, PhD, of University College London in the UK and cofounder of the AGORA Initiative, which aims to tackle the ...

In a clinical trial, vutrisiran was superior to placebo in reducing cardiovascular events and all-cause mortality in patients with ATTR-CM. The US Food and Drug Administration (FDA) has approved ...

Bone marrow flow cytometry is one of the most efficient ways to diagnose or exclude PNH. A patient with a history of cortical vein thrombosis with new-onset pancytopenia was diagnosed with paroxysmal ...

With the approval of the indication extension, pegcetacoplan now stands as the first C3 inhibitor for first-line PNH treatment. The European Commission (EC) has approved the indication extension of ...

The researchers used the Paris criteria to reach a final diagnosis. A team of international researchers presented the rare case of a male patient with autoimmune hepatitis-primary biliary cholangitis ...