MeSH tree: C20.673.795
The U.S. Food and Drug Administration has approved Andembry (garadacimab-gxii) as the only treatment targeting factor XIIa for prophylactic use to prevent attacks of hereditary angioedema (HAE).
Jun 21, 2025
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A new study from The University of Texas at Arlington details a novel strategy for how the body clears out dead cells during stress, revealing unexpected roles for well-known stress-response genes—a discovery that could help ...
Jun 9, 2025
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A firm handshake between proteins on immune cells is important for the body's ability to fight infection. Meanwhile, a weak grip helps explain the poor immune deficiencies caused by a rare genetic disorder.
Dec 10, 2024
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RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic disease.
Early results from a study of newborn screening methods show that DNA analysis picks up many more preventable or treatable serious health conditions than standard newborn screening and is favored by most parents who are offered ...
Oct 24, 2024
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A single treatment with a CRISPR-Cas9 based gene editing therapy is enough to replace the daily medication of patients with hereditary angioedema (HAE), a condition characterized by severe, painful and sudden onset of swelling, ...
Oct 24, 2024
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A study, published in the journal MedComm and led by Dr. Panpan Jiang and collaborators at Tongji Medical College, explores the critical role of the dedicator of cytokinesis 8 (DOCK8) gene in the differentiation of helper ...
Oct 9, 2024
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Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden's rare, inherited immune deficiency. But it's not available to him.
Jun 21, 2024
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For patients with hereditary angioedema, donidalorsen reduces the attack rate, according to a study published online May 31 in the New England Journal of Medicine to coincide with the annual congress of the European Academy ...
Jun 4, 2024
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For patients with type 1 or type 2 hereditary angioedema, sebetralstat provides faster times to beginning of symptom relief than placebo, according to a study published online May 31 in the New England Journal of Medicine ...
Jun 4, 2024
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