The majority (79%) of cardiac amyloidosis cases were responsible for sudden cardiac death. Cardiac amyloidosis can be associated with sudden cardiac death and is often undiagnosed during a person’s ...

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 ...

The recommendations were based on the available evidence, its certainty, and key judgments using the Grading of Recommendations Assessment, Development and Evaluation framework. New guidelines ...

There were 3 unmet needs in CIDP including the lack of specific biomarkers for the disease, weighing the relative value of various metrics, and understanding the optimal timing and approach to assess ...

In the pivotal LUNA 3 trial, durable platelet response at week 25 was achieved by 23% of patients with rilzabrutinib compared with 0% with placebo. The US Food and Drug Administration (FDA) has ...

Results from a 2-sample Mendelian randomization analysis showed that there was a significant association between primary biliary cholangitis (PBC) and osteoporosis. Primary biliary cholangitis (PBC) ...

The updated 2025 BSR management recommendations for patients with AAV provide systematic and evidence-based recommendations to support UK clinicians in managing AAV across the entire life course.

In addition to a 69.4% CR rate, the team observed good tolerability of the R2-MTX regimen, suggesting its suitability for older or less fit patients. High-dose methotrexate (HD-MTX) with lenalidomide ...

Although treatment was started with rituximab upon the CAD diagnosis, she died within 5 days. A case has been reported of a patient with a history of Raynaud’s phenomenon who was hospitalized due to ...

Speakers at the panel “Unlocking the future: the convergence of AI, personalized medicine, and genomics” at the World Orphan Drug Congress USA 2025 (Photo by Riya Ajmera) Artificial intelligence could ...

Nearly half of DEC-C-Ven–treated patients proceeded to hematopoietic stem cell transplantation vs only 16% of those treated with DEC-C. The addition of venetoclax to oral decitabine-cedazuridine ...

Rilzabrutinib significantly improved fatigue scores and reduced the need for rescue therapy by 52% vs placebo. Rilzabrutinib has shown clear evidence of efficacy and safety in patients with persistent ...