Attack severity in hereditary angioedema: a qualitative study to inform future economic evaluations.
Hereditary angioedema (HAE) is a rare condition with unpredictable, potentially life-threatening swelling attacks. Severity classifications in economic evaluations of new treatments often rely on parameters such as attack duration or ADL limitations, potentially oversimplifying patient experiences a…
Long-term prophylactic treatment and related real-world outcomes of patients with hereditary angioedema in Germany: A retrospective analysis of German claims and medical chart data.
Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent swelling episodes that are potentially life threating and substantially impair quality of life. This study aimed to generate real-world evidence on the utilization of long-term prophylaxis (LTP), residual disease burde…
Symptoms, Triggers, and Prodromes of Hereditary Angioedema in the Pakistani Population.
This cross-sectional observational study was conducted at the Department of Immunology, Armed Forces Institute of Pathology/ CMH / NUMS, Rawalpindi, Pakistan, from September 2021 to December 2024. It aimed to identify the frequency of common presentations, triggers, and prodromes of hereditary angio…
Donidalorsen for the treatment of hereditary angioedema.
Hereditary angioedema (HAE) is a rare disorder with a prevalence of approximately 1 in 50,000. Management of HAE brings challenges including the frequency of delivery of the medication, routes of medication delivery, cost of treatment and significant impact on quality of life for patients with HAE.…
Garadacimab in Hereditary Angioedema: Modeling Suggests Sustained Improvements in Attack-Free Probability and Quality of Life Over 5 Years.
Hereditary angioedema (HAE) attacks are recurrent, debilitating, and potentially life-threatening. Across the clinical program, garadacimab long-term prophylaxis demonstrated early onset and durable, sustained protection against HAE attacks and clinically meaningful improvements in health-related qu…
Genome editing for hereditary angioedema: A landmark trial and important unanswered questions.
Hereditary angioedema is a bradykinin-mediated genetic disorder increasingly controlled with effective long-term prophylaxis. In a phase 3 trial, a single dose of lonvoguran ziclumeran, an in vivo CRISPR-Cas9 therapy targeting KLKB1, significantly reduced attack rates. This approach represents a hig…
Beyond the Index Case: A Practical Framework for Cascade Family Screening in Hereditary Angioedema.
Hereditary angioedema (HAE) is a genetic disorder characterised by recurrent cutaneous or submucosal oedema. Most cases are caused by pathogenic SERPING1 variants (types I and II), while a smaller subset presents with normal C1-inhibitor (C1-INH) levels and function (HAE-nC1INH). Despite its potenti…