Forest Ray PhD,  —

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Pharming Group has committed to building a new manufacturing facility in the Netherlands to expand production of Ruconest (conestat alfa), its treatment for acute attacks of hereditary angioedema (HAE). Construction will begin in mid-2021. Once finished, it will create at least 40 new jobs, the company said. “With an…

Icatibant injection, a generic form of Firazyr from Fresenius Kabi, is now available to adults in the United States for the treatment of acute attacks of hereditary angioedema (HAE), the company announced. “For people with HAE, an acute attack can occur anywhere, at any time and…

Takeda Canada announced reaching an agreement for public funding in Canada of Takhzyro (lanadelumab), its treatment for eligible hereditary angioedema (HAE) patients. The agreement was reached between the company and the pan-Canadian Pharmaceutical Alliance, the agency responsible for setting the prices of brand and generic medications available throughout…

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…

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…

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…

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…