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 ...

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 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 ...

In the ADAPT SERON trial, treatment with efgartigimod led to rapid, significant, and sustained improvements in symptoms such as speech, vision, swallowing, and physical function. The US Food and Drug ...

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 ...

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 ...

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 ...

In a cross-sectional study, the researchers reported psychiatric comorbidities in 46.5% of 43,270 adults with neurofibromatosis. The most common psychiatric comorbidities included mood disorders (22.1 ...

Mutant uromodulin likely drives tubular injury that secondarily leads to glomerulosclerosis rather than primary podocyte disease. This case underscores that recognition of a genetic etiology can avert ...

Among those with ATTR, the sodium-glucose cotransporter 2 inhibitor group saw a nearly 50% reduction in all-cause mortality compared with controls (9.43% vs 19.2%; HR, 0.52). Sodium-glucose ...

The responder rate was 57%, and five patients (36%) achieved complete seizure freedom. The addition of stiripentol to standard-of-care therapies may offer meaningful seizure reduction for patients ...

If approved, CAM2029 could offer a self-administered alternative to intramuscular long-acting somatostatin analogs, with the potential to reduce treatment burden. The US Food and Drug Administration ...