Genetics

Gene editing offers hope for people with hereditary disorder

A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher of a trial published in the New England Journal ...

Diseases, Conditions, Syndromes

CRISPR treatment slows swelling in hereditary angioedema patients

Hereditary angioedema (HAE) is a rare, genetic disorder characterized by severe, recurring, and unpredictable swelling attacks in various organs and tissues of the body, which can be painful, debilitating, and life-threatening. ...

Genetics

New understanding of mysterious 'hereditary swelling'

For the first time ever, biomedical researchers from Aarhus University, Denmark, report cellular defects that lead to a rare disease, hereditary angioedema (HAE), in which patients experience recurrent episodes of swelling ...

Genetics

New hope for rare disorder

Hereditary angioedema is a chronic disorder that can sometimes be life-threatening. Now, a new drug therapy has been successfully tested in an international study headed by the University Hospital Frankfurt.

Diseases, Conditions, Syndromes

C1 inhibitor use reduces attacks in hereditary angioedema

(HealthDay)—Prophylactic use of the subcutaneous C1 inhibitor CSL830 is associated with a reduction in the frequency of acute attacks in patients with hereditary angioedema, according to a study published in the March 23 ...

Diseases, Conditions, Syndromes

In rare disorder, novel agent stops swelling before it starts

An early-stage clinical trial has found that, compared to a placebo, a novel medication significantly reduces potentially life-threatening episodes of swelling of the airway as well as the hands, feet, and abdomen of patients ...

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