Patricia Inácio, PhD, science writer —

Pharma and patient organizations came together this year in celebration of the 10th annual HAE Awareness Day — set aside to call global attention to hereditary angioedema (HAE). Set annually on May 16, this event aims to increase awareness among the public, healthcare professionals, healthcare decision-makers, and industry…

Haegarda is now available to adolescent and adult hereditary angioedema (HAE) patients across Canada for the prevention of HAE attacks, according to CSL Behring, the therapy’s developer. The company announced its preventive treatment, made available for eligible HAE patients in April, can now be given in…

Japanese researchers have identified a new mutation in the SERPING1 gene linked to hereditary angioedema (HAE) in a 27-year-old woman with a history of recurrent swelling attacks. Her case was described in a report, “Hereditary angioedema with a novel mutation, c.1481G>C, in the SERPING1 gene,” published…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

BioCryst Pharmaceuticals is seeking the approval of Orladeyo (berotralstat) in the United Kingdom as a preventive treatment for sudden and recurrent swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. If granted, Orladeyo would become the first oral therapy — found in trials to be…

Subcutaneous (under-the-skin) administration of human plasma-derived C1 inhibitor is an effective and safe option for managing hereditary angioedema during pregnancy and lactation, a case report suggests. The study, “Subcutaneous C1‐Inhibitor Concentrate for prophylaxis during pregnancy and lactation in a patient with C1‐INH‐HAE,” was published in the…

PHA121, an investigational oral therapy for hereditary angioedema attacks, was well tolerated across multiple doses in a group of healthy volunteers in a Phase 1 trial. Study data also indicates that PHA121 reached therapeutic levels in the first day of a 10-day treatment period, and stable…

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.