The term muscular dystrophy encompasses a group of genetic diseases characterized by progressive muscle weakness and wasting. There are different types of muscular dystrophy depending on genetic ...

Assessing Epidemiological Data Points The findings from this study echo that of another by Trivella and colleagues, also published in Hepatology Communications. This 2023 study is a literature review ...

Sickle cell disease (SCD) is an inherited blood disorder marked by defective hemoglobin. It causes red blood cells (RBCs) to deform into a sickle shape, which results in premature death of the RBCs, ...

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A patient with a history of cortical vein thrombosis with new-onset pancytopenia was diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) and received iptacopan, according to a study published in ...

Chronic lymphocytic leukemia (CLL) is a hematologic malignancy characterized by the clonal expansion of abnormal B lymphocytes. 1 Despite significant advancements in targeted therapies like Bruton’s ...

The first case of sickle cell disease (SCD) in the United States was described in 1904 when Walter Clement Noel, a 20-year-old dental student from Grenada, sought care for anemia at Chicago ...

A patient with a 10-year history of relapsing chronic lymphocytic leukemia (CLL) developed amyotrophic lateral sclerosis (ALS) and a drug-related sarcoid-like reaction (SLR). This case report was ...

The use of some patient-reported, disease-specific quality of life (QoL) scales in hereditary angioedema (HAE) are moderately recommended, according to a study published in Health and Quality of Life ...

As of November 2024, approximately 156 clinical trials for Duchenne muscular dystrophy (DMD) are ongoing or pending: 16 trials not yet recruiting, 65 currently recruiting, 28 trials that are active ...

Gastrointestinal (GI) symptoms are the second most frequent clinical manifestation of systemic mastocytosis (SM), after itching. They affect 15% to 85% of patients and have a significant impact on ...

Cold agglutinin disease (CAD) is a rare condition that is found in association with 15% of cases of autoimmune hemolytic anemia (AIHA). Hemolysis mediated by cold agglutinins results in mild to ...