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 ...

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 ...

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 ...

Taylor Kane is executive director of Remember the Girls, a nonprofit group that advocates on behalf of females with X-linked disorders (Photo courtesy of Taylor Kane) New Jersey native Taylor Kane ...

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 ...

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 ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Herwig Lange, MD, a neurologist who has been researching Huntington disease (HD) since 1969. Dr. Lange, a proponent of nondrug ...

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 ...

Obeticholic acid, seladelpar, and elafibranor all significantly increased the biochemical response rate. Elafibranor seems to be slightly more effective than seladelpar as a second-line therapy in ...

Concizumab-mtci works by inhibiting the tissue factor pathway inhibitor. This, in turn, enhances thrombin production and improves blood clotting. Concizumab-mtci (Alhemo ®) is the first subcutaneous ...

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 ...