Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare Artist contest, hosted by the EveryLife Foundation for Rare Diseases to heighten awareness about rare disease communities and salute the creativity of its members. Awardees will be able to display their art and…
Treatment with Takhzyro (lanadelumab) significantly improves health-related quality of life in people with hereditary angioedema (HAE), new data from its pivotal clinical trial show. While all dose regimens improved patient outcomes, the approved dose — 300 mg given every two weeks — provided the greatest benefits. Patients on this…
A case report describes a man with confirmed COVID-19 who developed acute angioedema — a skin symptom that had not previously been associated with the virus — in his lips. “To our knowledge, there has not been…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
The vast majority of people with hereditary angioedema (HAE) stress the importance of their preventative therapy correcting C1 inhibitor protein deficiency, allowing for fewer attacks, as the leading factor in evaluating a prophylactic treatment, according to a survey by The Harris Poll.
Assessing C1-inhibitor (C1-INH) activity for the diagnosis and monitoring of hereditary angioedema (HAE) can be done through three distinct validated methods with similar accuracy, a new study found. The study, “Parallel comparison of three methodologies for measuring functional C1-inhibitor in Hereditary angioedema patients,” was published in …
Pharming Group has committed to building a new manufacturing facility in the Netherlands to expand production of Ruconest (conestat alfa), its treatment for acute attacks of hereditary angioedema (HAE). Construction will begin in mid-2021. Once finished, it will create at least 40 new jobs, the company said. “With an…
Just a couple of weeks after its U.S. approval, Orladeyo (berotralstat) is now available for direct shipment to hereditary angioedema (HAE) patients in the United States who hold a prescription for the oral medication. According to BioCryst Pharmaceuticals, the maker of Orladeyo, the medicine will be provided exclusively…
Takeda’s Takhzyro (lanadelumab) has been approved in China for the routine prevention of hereditary angioedema (HAE) attacks in patients ages 12 and older, the company announced. It now becomes the “first approved modern therapy” available in the country for this indication, Takeda said in a press release.
PHA121 (PHA-022121), Pharvaris‘ oral therapy for hereditary angioedema (HAE), outperformed Firazyr (icatibant acetate) — an approved therapy with a similar mechanism of action — in a small study of healthy volunteers, the company announced in a press release. That…
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