At week 52, 50.9% of patients on elafibranor met the primary endpoint of biochemical response compared with 3.8% of those receiving the placebo. Lower baseline alkaline phosphatase (ALP) levels were ...

The FDA granted Fast Track Designation to coramitug for ATTR-CM as it advances in the ongoing phase 3 CLEOPATTRA trial. The US Food and Drug Administration (FDA) has granted Fast Track Designation to ...

Researchers developed an AI-based framework that can generate a report like a primary care physician, which can then help identify undiagnosed patients with ATTR-CM and individuals who are at risk of ...

The prevalence of irregular red cell alloantibodies was 8.04% in pediatric patients, with anti-E and anti-Mi antibodies the most common. Pediatric patients can develop irregular, non-ABO red cell ...

There were 7 cases of PAH temporally associated with bevacizumab in the French Pulmonary Hypertension Registry, but most had predisposing conditions. Bevacizumab exposure does not seem to lead to the ...

Findings from receiver operating characteristic analysis identified transferrin cutoff values of 194.0 mg/dL for differentiating active AAV from remission AAV and 259.5 mg/dL for distinguishing those ...

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 ...

Sparsentan is believed to help protect the kidneys and reduce damage through a mechanism that targets both endothelin A and angiotensin II receptors. The US Food and Drug Administration (FDA) has ...

The novel method, based on mass spectrometry, can simultaneously detect 35 HPA systems, 5 HNA systems, and CD36 polymorphisms. Researchers developed a high-throughput genotyping method that can ...

Responses proved durable, with 60% of patients maintaining an overall response at week 24. Daratumumab produced rapid and sustained platelet responses in adults with immune thrombocytopenia (ITP), ...

The streamlined machine learning model had a sensitivity of 90%, a specificity of 61%, and an F1 score of 83.3%. A streamlined machine learning model performs comparably to the Lasso plus random ...

Patient-reported outcomes reflected strong acceptance, with a median satisfaction score of 8.9 out of 10 on the Self-Injection Assessment Questionnaire. The zilucoplan (Zilbrysq ®) auto-injector ...