Investigational Therapy Earns Orphan Drug Designation for Hereditary Angioedema
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Investigational Therapy Earns Orphan Drug Designation for Hereditary Angioedema

According to a story from Business Wire, the biopharmaceutical company Astria Therapeutics, Inc., recently announced that its experimental therapy navenibart has earned the US Food and Drug Administration's Orphan Drug…

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Experimental Treatment Sebetralstat Shows Promise in Phase 3 HAE Study
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Experimental Treatment Sebetralstat Shows Promise in Phase 3 HAE Study

Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn't just a mere…

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Deucrictibant Reduces Monthly HAE Attacks by 84.5% in Phase 2 Study
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Deucrictibant Reduces Monthly HAE Attacks by 84.5% in Phase 2 Study

  Last year, the FDA placed a clinical hold on an Investigational New Drug (IND) application for deucrictibant for the on-demand and prophylactic treatment of individuals living with hereditary angioedema…

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Positive Phase 1 Data Available on Sebetralstat for HAE
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Positive Phase 1 Data Available on Sebetralstat for HAE

  According to a late October 2022 news release from pharmaceutical company KalVista Pharmaceuticals, Inc. (“KalVista”), the company released positive data from a Phase 1 study evaluating oral sebetralstat for…

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Company Halts Development of Hereditary Angioedema Drug Following Safety Issues
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Company Halts Development of Hereditary Angioedema Drug Following Safety Issues

According to a story from BioPharma Dive, KalVista Pharmaceuticals has halted its clinical trial evaluating an investigational therapy in development for hereditary angioedema. The trial had included a total of…

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Donidalorsen Reduces HAE Attacks, Study Shows
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Donidalorsen Reduces HAE Attacks, Study Shows

In patients with hereditary angioedema (HAE), the C1-inhibitor blood protein does not function properly, allowing bradykinin peptides to build up in the body. These then cause inflammation and fluid leakage;…

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ERN-RITA Enables Virtual Health Consultations With European Experts
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ERN-RITA Enables Virtual Health Consultations With European Experts

Note: this story was originally published by HAE Junior, a Patient Worthy partner Interview with Malena Vetterli from the European Reference Network (ERN-RITA) for rare immunodeficiencies, autoinflammatory and autoimmune diseases.…

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HAE Junior’s Drawing Exhibition Unveiled the Dreams and Ambitions of Children Living With Hereditary Angioedema

HAE Junior’s drawing exhibition unveiled the dreams, wishes, and ambitions of children & teenagers living with hereditary angioedema (HAE). The patient organization HAE Junior organized a drawing exhibition named We…

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Experimental Hereditary Angioedema Treatment Performs Well in Phase 2 Trial
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Experimental Hereditary Angioedema Treatment Performs Well in Phase 2 Trial

According to a story from PR Newswire, the biotechnology company Ionis Pharmaceuticals recently announced promising findings from its phase 2 clinical trial. This clinical trial was evaluating the company's experimental…

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