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),…

The levels of certain immune molecules called cytokines in the blood are better at identifying the types of specific autoantibodies present in ANCA-associated vasculitis (AAV) patients than at determining clinical diagnosis, a recent study shows. Based on this finding, researchers suggest that AAV patients should be classified by autoantibody type…

Patients with ANCA-associated vasculitis (AAV) who receive Rituxan (rituximab) infusions for maintaining sustained remission fare better in the long term than those receiving the immunosuppressant azathioprine, according to French researchers. Fewer patients who received Rituxan as remission-maintenance therapy experienced a relapse during a 60-month (five-year) follow-up. Researchers found that survival…

Prolonging immunosuppressive use may help patients with severe ANCA-associated glomerulonephritis —inflammation of the kidney — who still need dialysis after a standard three-month treatment, a study suggests. The study, “Late restoration of renal function in patients with severe ANCA-associated glomerulonephritis who were dialysis-dependent at presentation,” was published…

A new patient-reported outcome (PRO) questionnaire for ANCA-associated vasculitis (AAV) is a reliable and feasible tool able to distinguish between different disease states, according to a new study. The study, “Validation of the ANCA-associated vasculitis patient-reported outcomes (AAV-PRO) questionnaire,” appeared in the journal Annals of the…

Patients with ANCA-associated vasculitis (AAV) who develop pulmonary bleeding as a result of their condition fare significantly better if they receive plasma exchange therapy, a kind of blood purification technique, researchers say. Their study, “Effect of plasma exchange on in-hospital mortality in patients with pulmonary hemorrhage secondary to…

A rare case of granulomatosis with polyangiitis (GPA) affecting a thin layer of the abdomen — with no detectable anti-neutrophil cytoplasmic antibodies (ANCA) — was reported by researchers at Mitsui Memorial Hospital in Tokyo, Japan. The case report “Granulomatosis with Polyangiitis Localized in the Greater Omentum” was…

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”…

The Vasculitis Foundation announced the 2018 awardees of its one-year grants, amounting a total of $160,000. The three project winners were selected from 20 applications from research teams around the world. To date, the Vasculitis Foundation has granted more than $2.5 million for a total of 49 research studies. Among…