The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
News
Extracellular vesicles, or the tiny membrane-bound particles that are released from cells, could be used as a blood biomarker of disease activity in people with ANCA-associated vasculitis (AAV), a new study suggests. The study, “Microparticles expressing myeloperoxidase as potential biomarkers in anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitides…
The complement system, a part of the immune system, is overactive in people with active ANCA-associated vasculitis (AAV), but achieving remission appears to lower most of its components to normal levels, a study reports. The study, “The alternative complement pathway in ANCA‐associated vasculitis: further evidence…
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.
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