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 ...

Large v-waves with an amplitude of 10 mm Hg or more were a determinant of all-cause mortality and rehospitalization due to heart failure events in patients with wild-type ATTR-CM. Elevated v-wave ...

Sickle cell disease is inherited in an autosomal recessive pattern, which means that a child is born with SCD only when they inherit 2 defective copies of the sickle cell gene (1 from each parent). If ...

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 ...

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 ...

Intravenously administered zaltenibart was shown to be safe and effective in phase 2 clinical trials. Enrollment is under way to recruit patients for a phase 3 study designed to assess intravascular ...

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 ...

Results showed a significantly higher prevalence of liver disease among patients with chronic pruritus compared to healthy controls. Patients with liver diseases of different etiologies, including ...

Researchers attributed the findings to interactions between polyglutamine (poly-Q) tracts in HTT and TDP-43, a protein commonly implicated in ALS. A previously unknown link between the Huntington ...

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 ...