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 ...

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 ...

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 ...

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 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 ...

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 ...

A pattern recognition approach takes into account factors like disease progression rate, whether the disease symptoms are symmetrical or asymmetrical, whether they are predominantly distal or proximal ...

Denecimig aims to help the body form blood clots more effectively by mimicking the role of FVIIIa and bridging factor IXa and factor X to restore thrombin generation and support normal clot formation.

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 ...

Frank Rivera, president of Stronger Than Sarcoidosis, speaks at the 2024 World Orphan Drug Congress USA in Boston, Massachusetts (Photo by Manuela Callari, PhD) Prolonged uncertainty about the future ...