There was a reduction in volume of involvement, cardiac pyrophosphate activity, native T1, and extracellular volume during a median of 369 days between scans in patients with ATTR-CM being treated.

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 ...

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 ...

CAD accounts for approximately 15% of AIHA cases 2; various sources indicate that anywhere from 7% to 25% of cases of AIHA are caused by CAD. 3 Secondary CAD occurs as a result of underlying ...

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 ...

A multidisciplinary approach is necessary for the deep phenotyping and proper clinical management of individuals with primary mitochondrial diseases. A large percentage of patients (60%) with primary ...

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 ...

Patients with PCNSL should be closely monitored for extracerebral relapses, including ocular. Primary central nervous system lymphoma (PCNSL) can recur as isolated ophthalmologic and isolated cerebral ...

The navenibart phase 3 program will consist of the ALPHA-ORBIT phase 3 trial and the long-term extension trial, which are designed to support registration globally. Global start-up activities ...

Timothy Craig, DO, has been researching hereditary angioedema for 30 years. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Timothy Craig, DO, a tenured professor of medicine, ...

Sickle cell disease is inherited in an autosomal recessive pattern, which means that a child is born with SCD only when they inherit 2 defective copies of the sickle cell gene (1 from each parent). If ...

A baseline bleeding risk of 7.3 bleeds per year was observed without any FIX, and targeting trough FIX levels between 10 and 20 IU/dL reduced bleeds to very low or eliminated them. In people with ...