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

There are several experimental therapies under investigation for the treatment of Fabry disease, including enzyme replacement therapy (ERT), substrate ...

Amyloid transthyretin cardiomyopathy (ATTR-CM) is a progressive and life-threatening heart disease that can lead to increased ventricular wall thickness, restrictive cardiomyopathy, and heart failure.

Are you a healthcare provider looking for information on Diffuse Large B-Cell Lymphoma Epidemiology? Read about it here.

The addition of a 4-biomarker panel to morphology significantly aids the classification of intrahepatic cholangiocarcinoma (iCCA) specimens.

Global consensus was achieved on actionable treatment goals and outcome measures for patients with generalized pustular psoriasis (GPP).

Targeted therapies for IgG4-RD provide a potential alternative to glucocorticoids, affording better control of disease along with fewer side effects.

The levels of sialylated N-glycans in the serum could be a novel biomarker in chronic inflammatory demyelinating polyneuropathy (CIDP), a study found.

New research suggests cumulative ambient light exposure may cause the underestimation of bilirubinometer readings among children with neonatal jaundice.

Two phase 3 clinical trials are recruiting patients with CIDP to evaluate the efficacy of riliprubart treatment.

A supratherapeutic dose of 1400 mg of vatiquinone is safe and well-tolerated in patients with Friedreich ataxia (FA), a study found.

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 ...