Population Pharmacokinetic/Pharmacodynamic Modeling of Donidalorsen, an Antisense Oligonucleotide in Development for Prophylaxis of Hereditary Angioedema. [PDF]
Diep JK +7 more
europepmc +1 more source
Characterization of missense variants in the signal peptide of C1 esterase inhibitor. [PDF]
Ren Z +4 more
europepmc +1 more source
Impact of Oral Sebetralstat on Anxiety Associated With Hereditary Angioedema Attacks. [PDF]
Craig T +24 more
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Garadacimab for the long-term prophylaxis of hereditary angioedema. [PDF]
Aygören-Pürsün E +5 more
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Post Hoc Analysis of Recombinant C1 Inhibitor Clinical Data Using Contemporary Endpoints for Hereditary Angioedema. [PDF]
Riedl MA +12 more
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Modeling Hereditary Angioedema With Personalized Expanded Potential Stem Cell-Derived Hepatocytes: A CRISPR-Validated Platform for Mutation-Specific Mechanisms and Therapeutic Innovation. [PDF]
Liu X +10 more
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A Treat-to-Target approach in hereditary angioedema: expert consensus from a European committee. [PDF]
Cancian M +10 more
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Donidalorsen for the Treatment of Hereditary Angioedema: A Review of Clinical Studies. [PDF]
Riedl MA +7 more
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Hereditary angioedema (HAE) is a rare autosomal dominant genetic disorder that usual results from a decreased level of functional C1-INH and clinically manifests with intermittent attacks of swelling of the subcutaneous tissue or submucosal layers of the respiratory or gastrointestinal tracts.
R Gentry, Wilkerson, Joseph J, Moellman
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