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Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

BioCryst Pharmaceuticals has licensed rights to Torii Pharmaceutical for the commercialization of BCX7353 — an oral, once-daily investigational therapy for the prevention of hereditary angioedema (HAE) attacks — in Japan, pending the treatment’s approval. The plan is for BioCryst to submit a Japanese new drug application (JNDA)…

Having systemic lupus erythematosus (SLE) can considerably increase the likelihood of developing angioedema, a new study suggests. Women, African-Americans, and younger patients may be particularly susceptible, its findings show. The research, “Systemic Lupus Erythematosus and Angioedema: A Cross-Sectional Study From the National Inpatient Sample,” was published in the…

Acquired angioedema can be the first symptom of underlying conditions such as lymphoma. In those cases, cancer treatment may be the best approach to stop angioedema manifestations, a case report suggests. The study, “A Case of Acquired Angioedema with Low C1 Inhibitor (C1-INH) Associated with…

Kalbitor (ecallantide) is a potential treatment for angioedema attacks that do not respond to medication, in adolescents who also have systemic lupus erythematosus (SLE), even if they have normal levels of C1 inhibitor protein, two case reports suggest. The study combining the reports, “Ecallantide:…

A genetic technique called allelic discrimination — which detects a specific variant of the F12 gene — might make diagnosing certain hereditary angioedema-causing mutations quicker and cheaper, a new study showed. Titled “A cost-effective algorithm for diagnosis of hereditary angioedema with normal C1 inhibitor:…

Short-term use of Ruconest (recombinant human C1 inhibitor) as a preventive treatment reduces the risk of hereditary angioedema (HAE) attacks triggered by medical and dental procedures or by stress, a case series reports. The study, “Recombinant Human C1 Esterase Inhibitor as Short-Term Prophylaxis…

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.