News

US Patients Underreport Burden of HAE to Their Doctors, Study Says

The burden that hereditary angioedema (HAE) has on patients’ quality of life is seldom discussed during clinical visits, a U.S. study suggests. The study also found that patients fail to fully describe disease symptoms during such appointments, so their struggles are not well-documented. Therefore, researchers recommend a more patient-driven…

Takeda Canada Seeks AI, Digital Solutions With Innovation Challenge

With a goal of finding new digital technologies and artificial intelligence (AI) solutions for patients with rare genetic disorders like hereditary angioedema (HAE), and for inflammatory bowel diseases, Takeda Canada has introduced an innovation challenge. Specifically, Takeda Canada is seeking new and breakthrough solutions in early diagnosis, as…

New Gene Mutation Linked to HAE Type 1 Identified in Spanish Family

A new mutation in the SERPING1 gene linked to hereditary angioedema (HAE) type 1 was identified in three of five members of a Spanish family. Results were published in a letter to the editor of the journal Pediatric Allergy and Immunology titled “Hereditary angioedema caused by a novel intronic variant of SERPING1.” HAE is a form of…

HAE Patients Have High Rate of Cancer, Canadian Study Suggests

People with hereditary angioedema (HAE) commonly have histories of cancer, which raises the possibility that it might predispose a person to develop cancer. These findings were reported in the study “Malignancy and immune disorders in patients with hereditary angioedema,” published in the journal Allergy, Asthma…

Pharming, Sanofi Extend Partnership to Manufacture Ruconest

Pharming Group and Sanofi have renewed an agreement for the manufacturing of Ruconest (conestat alfa), Pharming’s approved treatment for swelling attacks in people with hereditary angioedema (HAE). The two companies have been working together for more than a decade, and now have agreed to extend the collaboration…

Prepare to Light Up Buildings for Rare Disease Day 2022

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the U.S. sponsor for Rare Disease Day on Feb. 28. The annual awareness day spotlights approximately 7,000…

Trial of HAE Gene-editing Therapy NTLA-2002 Doses Its First Patient

The first participant has been dosed in a Phase 1/2 clinical trial that is testing Intellia Therapeutics’ gene-editing therapy, NTLA-2002, in people with hereditary angioedema (HAE). “With the progress of our first-in-human clinical study evaluating NTLA-2002 for people living with HAE, we look forward to beginning clinical testing as…