A5011446920- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Hemophilia Treatment and Research
- COVID-19 Clinical Research Studies
- Long-Term Effects of COVID-19
- Autoimmune Bullous Skin Diseases
- Blood Coagulation and Thrombosis Mechanisms
- SARS-CoV-2 and COVID-19 Research
- Vitamin K Research Studies
- Intensive Care Unit Cognitive Disorders
- Venous Thromboembolism Diagnosis and Management
- Chronic Myeloid Leukemia Treatments
- Enzyme function and inhibition
- Complement system in diseases
- Heparin-Induced Thrombocytopenia and Thrombosis
- SARS-CoV-2 detection and testing
- Respiratory Support and Mechanisms
- Mast cells and histamine
- Peptidase Inhibition and Analysis
- Urticaria and Related Conditions
University of Amsterdam
2022-2024
University Hospital Frankfurt
2024
Charité - Universitätsmedizin Berlin
2024
Fraunhofer Institute for Translational Medicine and Pharmacology
2024
Goethe University Frankfurt
2024
Centre National de la Recherche Scientifique
2024
Centre Hospitalier Universitaire de Lille
2024
Centre Hospitalier Universitaire de Grenoble
2024
Humboldt-Universität zu Berlin
2024
University of Auckland
2024
Hereditary angioedema is a rare genetic disease that leads to severe and unpredictable swelling attacks. NTLA-2002 an in vivo gene-editing therapy based on clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9. targets the gene encoding kallikrein B1 (
BackgroundHereditary angioedema is a rare genetic disease characterized by severe and unpredictable swelling attacks. NTLA-2002 an in vivo gene-editing therapy that based on clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR-associated protein 9. targets the gene encoding kallikrein B1 (KLKB1). A single dose of may provide lifelong control attacks.MethodsIn this phase 2 portion 1–2 trial, we randomly assigned adults with hereditary 2:2:1 ratio to receive 25 mg or 50...
Angioedema due to acquired C1-inhibitor deficiency is a very rare but serious disease, with an estimated prevalence of 1 per 500,000 persons. There are no approved therapies treat or prevent angioedema swelling in patients this condition. Deucrictibant specific, orally bioavailable, competitive antagonist the bradykinin B2 receptor currently under investigation for hereditary angioedema.
Abstract Background Hereditary angioedema (HAE) is a potentially fatal disease characterized by unpredictable, recurrent, often disabling swelling attacks. In randomized phase 2 study, donidalorsen reduced HAE attack frequency and improved patient quality‐of‐life (ISIS721744‐CS2, NCT04030598). We report the 2‐year interim analysis of open‐label extension (OLE) study (ISIS 721744‐CS3, NCT04307381). Methods OLE, on‐treatment period consisted fixed (weeks 1–13, 80 mg subcutaneously every 4...
Clinical trials investigating drugs for the acute treatment of hereditary angioedema attacks have assessed many different outcomes. This heterogeneity limits comparability trial results and may lead to selective outcome reporting bias a high burden on participants. To achieve consensus core set composed key outcomes that ideally should be used in all clinical efficacy involving attacks. We conducted Delphi study relevant parties: patients with angioedema, expert clinicians researchers,...
Hereditary angioedema (HAE) is characterized by recurrent mucosal and cutaneous swellings, resulting from excessive bradykinin generation, which the end product of kallikrein/kinin system.[1] [2] HAE predominantly occurs in patients with congenital C1-inhibitor deficiency. controls activation factor XII (FXII) plasma prekallikrein (PK). Notably, bradykinin-mediated also described normal levels functionality C1-inhibitor.[3] Inhibition PK currently being investigated as a prophylactic...
Clinical ImplicationsMore than one-third of patients with hereditary angioedema are hesitant to have children because their condition. Patients support the availability reproductive options for prevent birth an affected child, including preimplantation genetic testing. More Hereditary (HAE) is autosomal-dominant monogenic disorder, causing painful or hindering swellings that can even be life-threatening. The severity and frequency attacks differ between patients, regardless...
Daratumumab-based treatment could control severe, treatment-refractory, life-threatening angioedema due to acquired C1-inhibitor deficiency associated with monoclonal gammopathy.