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 ...

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 ...

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 ...

Yiannis Papazoglou is one of only 40 people in the world with a mutation of the IRF2BPL gene. Foundation Aims to Help Patients With Rare Diseases—Even Before They’re Recognized The Undiagnosed ...

Prophylactic treatment with cyclophosphamide against graft-versus-host disease following allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis (MF) is “extremely effective, ...

Although primary intraosseous Rosai-Dorfman disease (RDD) may be characterized by fibrosis and an increase in Ig4+ plasma concentration, it does not meet the diagnostic criteria for immunoglobulin ...

A CRISPR/Cas9-modified Janus kinase 2 (JAK2) V617F model may be useful to develop new therapies against myeloproliferative neoplasms (MPNs), showed a new study published in the International Journal ...

Adult patients with Fabry disease have a lower lean mass compared to healthy people, according to a new study published in the Journal of Rare Diseases. The accumulation of lyso-Gb3 is also correlated ...

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 ...

Which of the statements below about the complications of immunoglobulin G4-related disease (IgG4-RD) are correct? Select one of the options for each question, then submit your answers to see how well ...

Individuals with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) undergoing treatment with fludarabine, cyclophosphamide, and rituximab (FCR) are at increased risk for other ...

A risk of pregnancy complications and adverse birth outcomes has been observed in mothers with alpha-1 antitrypsin deficiency (AATD), according to a study recently published in Plos One. The ...