News

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

KalVista Pharmaceuticals is planning to initiate a Phase 2 trial of its investigational oral therapy KVD824 as a preventive treatment for hereditary angioedema (HAE) attacks, the company announced. The decision follows promising data from two clinical trials, in which KVD824 was well tolerated and reached therapeutically relevant…

A single dose of NTLA-2002, Intellia Therapeutics’ investigational gene-editing therapy for hereditary angioedema (HAE), induces sustained reductions in the activity of plasma kallikrein — a protein involved in HAE swelling attacks — for at least 11 months, preclinical data shows. Based on these promising data in non-human…

In cases where targeted treatment for hereditary angioedema (HAE) is not available for rapid management of swelling attacks, patients may benefit from fresh frozen plasma (FFP) therapy, according to a recent case in Pakistan. Despite little information regarding best practices when using FFP, this success points to the…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions to…

The U.S. Food and Drug Administration (FDA) has extended the use of Haegarda, a C1-INH replacement medication, as a routine treatment to prevent hereditary angioedema (HAE) attacks in children ages 6 and older. Haegarda, a prophylactic treatment available as an under-the-skin (subcutaneous) injection that can be taken at…

Preventive treatment with Haegarda, an under-the-skin C1-INH replacement medication by CSL Behring, significantly lowers the number of swelling episodes experienced by children and adolescents with hereditary angioedema (HAE), an analysis of the COMPACT trial’s open-label extension study found. The treatment caused a 97% reduction in HAE…

Bridge Medicines and The Rockefeller University are collaborating to treat hereditary angioedema (HAE) by developing a series of compounds to inhibit the coagulation factor XII, known as FXIIa. Overactive FXIIa — often caused by a mutation in the F12 gene — is the leading cause…

The Phase 2 clinical trial testing KalVista Pharmaceuticals‘ oral investigational therapy KVD900 as an on-demand treatment for hereditary angioedema (HAE) attacks has completed enrollment, the company announced. Results from the trial are expected by year’s end. Meanwhile, the company has submitted a pediatric investigational plan for KVD900 to the European Medicines…

Short-term preventive treatment with Berinert, a plasma-derived form of the C1 inhibitor protein, protects people with hereditary and acquired C1-inhibitor (C1-INH) deficiency from angioedema attacks after dental procedures, according to a recent study. The study, “Short-term prophylaxis in patients with angioedema due to C1-inhibitor…