News

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…

Plasma exchange does not prolong the survival of patients with severe ANCA-associated vasculitis connected with diffuse alveolar hemorrhage, a potentially life-threatening condition in which blood starts to build up in the lungs, a study has found. The results challenge the notion that plasma exchange therapy might improve…

The presence of detectable ANCA antibodies and B-cells after initial treatment with rituximab — used to induce remission in people with ANCA-associated vasculitis (AAV) — predicts a greater risk of relapse, a new study shows. The study, “Proteinase-3-anti-neutrophil cytoplasmic antibodies (PR3-ANCAs) predict relapses…

The complement system, a component of the immune system, is overactive in people with necrotizing glomerulonephritis, or inflammation in the tiny blood vessels of the kidneys, caused by ANCA-associated vasculitis (AAV), a study reports. Some complement proteins seem to correlate with more severe kidney disease in these…

Fingolimod (FTY720), a medication used to treat multiple sclerosis, also appears to ease many of the symptoms of ANCA-associated vasculitis (AAV), a new study in rats suggests. The study, “Sphingosine-1-phosphate receptor modulator FTY720 attenuates experimental myeloperoxidase-ANCA vasculitis in a T cell-dependent manner,” was published…

A combination of rituximab, plus glucocorticoids, is highly effective at re-inducing remission in patients with relapsed ANCA-associated vasculitis (AAV), according to a Phase 3 clinical trial. Trial findings were described in the study, “Rituximab as therapy to induce remission after relapse in ANCA-associated vasculitis,”…

ChemoCentryx is asking the U.S. Food and Drug Administration (FDA) to approve avacopan, a potential oral treatment for people with ANCA-associated vasculitis (AAV). The request, in the form of a new drug application (NDA), is based on data from the ADVOCATE Phase 3 trial (NCT02994927) in adult patients.

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

Blood levels of the soluble urokinase plasminogen activator receptor (suPAR) protein may be a helpful tool for assessing disease severity and prognosis in people with ANCA-associated vasculitis (AAV), a new study reports. The study, “The Plasma…

Low levels of the protein alpha-1-antitrypsin (AAT) do not appear to be enough to increase the risk of ANCA-associated vasculitis (AAV) in people with chronic obstructive pulmonary disease (COPD) — despite prior associations of AAT deficiency with AAV — according to a new study.