News

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…

Antisense oligonucleotides (ASOs) being developed by Ionis Pharmaceuticals may reduce the frequency of attacks in people with hereditary angioedema (HAE), a small study suggests. The study, “Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema,” funded by Amsterdam UMC, was published in The New England…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

A case report suggests that the body has multiple ways to produce bradykinin, a molecule that can cause angioedema, in patients taking ACE inhibitors. The report, “Lisinopril-Induced Angioedema in a Patient with Plasma Prekallikrein Deficiency,” was published in the peer-reviewed journal TH Open. Angioedema can be…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

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…