The prevalence of PBC is expected to rise in the United States and the wider world, requiring concrete, proactive action.

Diet and nutrition play a key role in managing Duchenne muscular dystrophy (DMD). Dietary goals include preserving lean muscle mass, preventing weight gain and fat accumulation, maintaining bone ...

The damage associated with IgG4-RD can be irreversible, and tools such as the IgG4-RD DI allow clinicians to better track disease progression.

Investigational gene therapy showed sustained clinical benefit and long-term safety in severe hemophilia B over 13 years of follow-up.

Researchers proposed a link between an increase in average factor activity level and lower ABR, as well as improved health-related quality of life HRQoL.

The appendicular lean mass was lower in patients with Fabry disease at a mean of 20,324 g compared to 25,529 g in healthy people.

Anti-contactin-1 (CNTN1) antibodies may be a novel biomarker for the diagnosis of chronic inflammatory demyelinating polyneuropathy (CIDP), a study found.

Neuromyelitis optica spectrum disorder (NMOSD) and multiple sclerosis (MS) have a negative impact on employment, a study found.

Bilirubin results may differ between neonates of different ethnicities, a possibility that should inform the interpretation of these results.

There is a substantial cardiovascular disease burden, particularly ischemic heart disease, among adults with sickle cell disease (SCD).

Levels of MCP-1, TNFR-2, YKL-40, and cystatin C in the serum may be potential biomarkers for different aspects of Fabry disease activity, a study found.

Sole reliance on AFP levels for identifying hepatocellular carcinoma (HCC) may be insufficient for rendering an accurate diagnosis.