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 ...

Test Your Knowledge About the Signs and Symptoms of FNAIT Test Your Knowledge About the Epidemiology of FNAIT Test Your Knowledge About the Diagnosis of FNAIT Rare Disease Advisor, a trusted source of ...

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 ...

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 ...

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 ...

There is a dichotomy in quality-of-life outcomes in patients with PAH, with significant physical impairments and relatively preserved mental health scores. Patient-reported outcome measures play a key ...

Nephrologist Gates Colbert, MD, speaks at the Renal Physicians Association 2026 Annual Meeting in Atlanta, Georgia (Photo by Riya Ajmera) With 4 pivotal therapy types now available, CKD remission is ...

Oral corticosteroids were the most common treatment used by patients with systemic autoimmune rheumatic disease-associated interstitial lung disease whose disease had progressed to PPF. A recent study ...