News

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

Self-administration of Berinert was found to be safe and effective and reduces the need for emergency room visits during acute attacks in patients with hereditary angioedema type 1, according to researchers. Their study, “…

Comparing the genetic profile of patients with hereditary angioedema during remission and in acute attacks, researchers identified two genes that seem to participate in edema formation. The genes – uPAR and ADM – are involved in vessel dilation and inflammation, and may be potential therapeutic targets for the disease. The…

A new strategy that uses small protein fragments with an unconventional structure may be used to target many different disease-related proteins, according to French researchers. The team at the Ecole Polytechnique Fédérale de Lausanne has already developed a molecule that targets kallikrein, the protein that causes hereditary angioedema, and another targeting…

The average yearly cost of care for hereditary angioedema (HAE) patients is $409,925 per person, with the cost of medications making up 97% of that total, Prime Therapeutics found in a new study. Based on the findings, Prime, a pharmacy benefit management company, is calling for the use of pharmacist…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

A marketing authorization application for Lanadelumab, Shire‘s investigational therapy for hereditary angioedema (HAE), was recently cleared by the Swiss Agency for Therapeutic Products (Swissmedic), the company announced. Supported by data from four clinical trials, acceptance of the marketing authorization application signals that the formal review process of the…

Patients who develop angioedema symptoms after receiving stroke medication, and are not responsive to first-line angioedema therapy, may benefit from Firazyr (icatibant), a recent case report suggests. “Icatibant for the treatment of orolingual angioedema following the administration of tissue plasminogen activator” was published in The American Journal…

Levels of complement proteins — proteins of the immune system — may predict disease activity in patients with hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE), according to Japanese researchers. Their study, “The relationship between complement levels and disease activity in Japanese family cases of hereditary angioedema with C1-INH…