Amyloid transthyretin cardiomyopathy (ATTR-CM) is a progressive and life-threatening heart disease that can lead to increased ventricular wall thickness, restrictive cardiomyopathy, and heart failure.

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 ...

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 ...

Patients with alpha-1 antitrypsin deficiency (AATD) receiving inhaled ATT therapy have demonstrated significant improvements in restoring protease–antiprotease homeostasis, according to results of a ...

A recent retrospective study highlighted the importance of human leukocyte antigen (HLA)-matched platelet units in improving outcomes for patients with immunized platelet transfusion refractoriness.

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 ...

The CSHA Clinical Frailty Scale (CFS) is a valuable tool for predicting treatment-related toxicity and discontinuation in older adults receiving targeted therapy for chronic lymphocytic leukemia (CLL) ...

Diffuse large B-cell lymphoma (DLBCL) is a type of cancer that originates in and affects the lymphatic system. 1 It is the most common type of non-Hodgkin lymphoma (NHL), accounting for approximately ...

The administration of ARGX-119 led to improved survival and muscle function in a passive transfer myasthenia gravis (MG) mice model; however, the efficacy appears to be limited to a subset of patients ...

A strong association has been observed among patients with systemic mastocytosis (SM) who harbor the somatic missense KIT p.D816V variant in peripheral blood leukocytes and severe sting anaphylaxis. A ...

A male patient initially suspected of having immunoglobulin G4-related disease (IgG4-RD) had his diagnosis revised to Castleman disease following further investigations. This case report was published ...

Treatment with risdiplam over 30 months led to overall clinically meaningful improvement in most adult patients with spinal muscular atrophy (SMA) type 2 or 3, according to a single-center ...