Patients with ATTR-CM had significantly impaired global longitudinal strain and higher mechanical dispersion compared to their first-degree relatives who were not affected by the disease. Increased ...

Several factors can contribute to the suppression of bone marrow erythropoiesis in infants with HDFN, including maternal antibodies, intrauterine transfusion, and simple transfusion. The suppression ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

My recent liver biopsy was a case study of Murphy’s Law in action. And as I share the details of my saga, I’m mindful that my experience is the exception, not the rule. I do not want to discourage ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Brian O’Mahony, president of the Irish Haemophilia Society and one of the few patients in Europe with hemophilia B to receive ...

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 ...

Between 1970 and 1995, the incidence of new NHL cases increased. Between 2007 and 2016, new cases declined by approximately 0.9% per year, and mortality from NHL decreased by 2.2% per year. 3 ...

In a nonhuman primate model of acquired hemophilia A, treatment with PS-siRNA 40031 restored thrombin generation to normal levels. Reducing protein S (PS) levels can increase thrombin generation and ...

Immunohistochemistry analyses detected a correlation between increased CDK1 expression and higher levels of LDH, a key indicator of greater disease severity in DLBCL. Higher levels of CDK1 expression ...

Patients with early-stage PBC and ductopenia had reduced biochemical response to ursodeoxycholic acid treatment. Ductopenia is a significant risk factor for worse biochemical profiles and poor ...

Emerging therapies such as bezuclastinib and elenestinib show promise in improving disease control with potentially fewer toxicities. Other novel strategies, such as immune-based therapies, are under ...

The potential of axonal damage as a biomarker of disease severity and predictor of further outcomes is highlighted by its effect on functional disability, even in early disease stages. Patients who ...