News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

BioCryst Pharmaceuticals is prepared to launch Orladeyo (berotralstat), an oral treatment for hereditary angioedema (HAE), by the end of the year if the medication is approved, the company has announced. “We are currently in an exciting transformation from a company primarily focused on R&D [research and development]…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

More insight and study is needed into the symptoms, proper management, and potential complications of pregnancy-related hereditary angioedema (HAE) attacks, according to a case report of a woman who is now in her third pregnancy. In this case, abdominal pain appeared as the woman’s main symptom, and her exacerbations…

KalVista Pharmaceuticals anticipates beginning a Phase 2 clinical trial in the second half of this year to test KVD824, an investigational preventive treatment for hereditary angioedema (HAE). In addition, the results of a Phase 2 clinical trial of KVD900, being investigated as an on-demand treatment for…

HAE International, a global nonprofit that supports local hereditary angioedema (HAE) patient groups and organizations, has expanded their emergency card template to a total of 34 languages — and plans to add more in the future. The emergency cards are a means for patients to quickly and…

Cipla has received final approval from the U.S. Food and Drug Administration (FDA) for a generic version of Firazyr (icatibant injection), an approved treatment for acute attacks in adults with hereditary angioedema (HAE). The company announced the FDA approval of its abbreviated new drug application for icatibant…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

Preventive treatment with Takhzyro (lanadelumab) significantly reduces the number of swelling attacks in people with hereditary angioedema (HAE), even in the early phases of treatment, a post hoc analysis from the HELP trial shows. The benefits are evident within two weeks of treatment and progressively increase over time, the researchers…

Regular Haegarda treatment in older people with hereditary angioedema (HAE) — those ages 65 and older — was well-tolerated and effective in stopping HAE-related attacks, a post-hoc analysis of the COMPACT Phase 3 clinical trial revealed. The therapy was found to be well-tolerated…