News

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

The National Institute for Health and Clinical Excellence (NICE) has recommended Takhzyro (lanadelumb) as a preventive treatment for hereditary angioedema (HAE) attacks in patients 12 and older, under certain conditions. NICE’s final appraisal determination (FAD) states that Takhzyro should be used only if: patients…

Prophylactic, or preventive treatment with Haegarda quickly reduced the number of attacks — sudden episodes of swelling — in people with hereditary angioedema (HAE) caused by C1-inhibitor deficiency, an exploratory analysis of a Phase 3 trial shows. The number of attacks were reduced regardless of baseline patient characteristics.

Pharvaris reported raising $66 million in a second round of financing, allowing the company to advance the clinical development of PHA121, its lead investigational oral therapy for hereditary angioedema (HAE). New investors include such life science and investment companies as  Foresite Capital together with …

Use of genetic testing enabled the detection of hereditary angioedema (HAE) in a previously misdiagnosed woman, found to be carrying a gene variant linked with HAE of unknown genetic cause. The findings support the consideration of genetic testing to diagnose unexplained angioedema, with an end goal of speeding treatment…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

Older patients with hereditary angioedema types 1 or 2 can as safely be treated for an acute attack with Firazyr (icatibant) as younger ones, a study found, even though people 65 or older tend to metabolize medicines differently and often are being treated for multiple disorders. The study, “…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Researchers have developed a new rat model of hereditary angioedema (HAE) that more closely mimics the swelling episodes experienced by humans, which might improve studies about the mechanisms that cause the disease. The model has been described in a new paper, “Endothelial B2-receptor overexpression as an…

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…