The majority (79%) of cardiac amyloidosis cases were responsible for sudden cardiac death. Cardiac amyloidosis can be associated with sudden cardiac death and is often undiagnosed during a person’s ...

Large v-waves with an amplitude of 10 mm Hg or more were a determinant of all-cause mortality and rehospitalization due to heart failure events in patients with wild-type ATTR-CM. Elevated v-wave ...

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 ...

Speakers at the panel “Unlocking the future: the convergence of AI, personalized medicine, and genomics” at the World Orphan Drug Congress USA 2025 (Photo by Riya Ajmera) Artificial intelligence could ...

A patient presented with task-related isolated transient weakness in her right hand, which started 15 years before and lasted for 9 years without worsening or improvement. Distal myasthenia gravis (MG ...

(L-R) Durhane Wong-Rieger, Cynthia Cassandro, Jida El Hajjar, Armelle Pindon and Richie Kahn participate in a panel discussion at the 2024 World Orphan Drug Congress USA. (Photo by Manuela Callari, ...

Benjamin Forred, director of translational research and the CoRDS Registry, Sanford Health, at the 2024 World Orphan Drug Congress USA. (Photo by Manuela Callari, PhD) The CoRDS registry, the largest ...

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 ...

In addition to a 69.4% CR rate, the team observed good tolerability of the R2-MTX regimen, suggesting its suitability for older or less fit patients. High-dose methotrexate (HD-MTX) with lenalidomide ...

Intravenous immunoglobulin-induced thrombocytopenia was reported in 146 cases, with the highest incidence seen in patients with immune-mediated skin diseases. Intravenous immunoglobulin (IVIg) therapy ...

More extensive investigation in ongoing and independent studies is warranted, with implications for donor registry, clinical, and laboratory practices. Among individuals who are refractory to random ...

A new Delphi consensus study involving patients with hereditary angioedema (HAE), clinicians, researchers, pharmaceutical companies, and regulatory entities has achieved consensus on a core set of 5 ...