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 ...

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 ...

The median alkaline phosphatase levels went down from 138 U/L to 85 U/L after 1 year of treatment with pemafibrate plus ursodeoxycholic acid. Pemafibrate plus ursodeoxycholic acid (UDCA) results in ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

91.7% of prognostic studies reported a significant association between bilirubin levels and outcomes. The levels of bilirubin and alkaline phosphatase, as well as composites including these 2, can be ...

The Reagan-Udall foundation was named for President Ronald Reagan and Congressman Morris “Mo” Udall from Arizona, each of whom had an incurable neurodegenerative disease. The Reagan-Udall Foundation ...