HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

ANCA-associated vasculitis (AAV) patients have lower levels of low-density lipoprotein (LDL), or “bad cholesterol,” when compared to people without the disease, a study suggests. These differences seemed to dissipate over time, which suggests a link between inflammatory status and LDL levels. Researchers advise more research is needed to…

Work productivity of people with ANCA-associated vasculitis is influenced by their disease activity, the extent of organ damage, and their health-related quality of life, a study reports. The findings were presented in a poster, titled “Association of Work Productivity Assessed by Absenteeism and Presenteeism with Disease Activity, Damage…

Avacopan (CCX168) can effectively improve remission rates, kidney function, and overall quality of life of patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), compared with standard therapy with steroids, according to top-line results from a pivotal Phase 3 clinical trial.   ChemoCentryx and its collaborator, Vifor…

Being a former or current cigarette smoker significantly increases the risk of developing ANCA-associated vasculitis (AAV), data from a large U.S. study show. This association was particularly  strong in patients with antibodies against myeloperoxidase (MPO). The abstract with the findings, titled “Cigarette Smoking Is a…

High-dose glucocorticoids — a type of corticosteroid hormone — are commonly prescribed as part of remission-induction therapy to people with ANCA-associated vasculitis (AAV) in Europe, a retrospective study reports. The majority of the patients remained on glucocorticoids for over a year, but only a minority reached full…

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

People with systemic lupus erythematosus (SLE) or granulomatosis with polyangiitis (GPA) who undergo kidney transplants have high survival rates over the long term without evidence of an increased risk of cancer, a study has found. Titled “Long-Term Outcomes of Renal Transplant in Patients With End-Stage…

Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis can result in inflammation of the appendix — commonly known as appendicitis — without any additional systemic symptoms, researchers report for the first time. The finding highlights the diverse effects of this autoimmune disorder and the importance of careful patient evaluation for adequate diagnosis…

Dialysis for Japanese patients with kidney disease triggered by ANCA-associated vasculitis led to fewer life-threatening relapses and reduced doses of immunosuppressive steroids, a long-term study suggests. The study, “Long‑term follow‑up characteristics of patients with anti‑neutrophil cytoplasmic antibody (ANCA)‑associated vasculitis (AAV) receiving chronic…