Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…
The use of radiation therapy on granulomatosis with polyangiitis (GPA) patients with cancer should be done with caution to avoid potential complications, a case report suggests. The study, “Lower cranial nerve palsy during radiotherapy for glottic cancer in a patient with Wegener’s granulomatosis: An interesting case report,” appeared in…
PredictImmune has expanded its intellectual propriety portfolio with a new patent approved in Canada covering the company’s methods for predicting the risk of progression among patients with autoimmune diseases, like ANCA-associated vasculitis (AAV) and inflammatory bowel disease (IBD). This follows the 2018 approval of previous patent applications…
G71.01 is, literally, the code for Duchenne muscular dystrophy.  Q93.51 stands for Angelman syndrome, and G40.419 covers generalized and treatment-resistant epilepsies, which groups like Orphanet and the American Epilepsy Society define as including Dravet syndrome. All three designations are among some 70,000 diseases listed in the latest…
A combination of belimumab plus standard maintenance treatment is no better than standard of care alone at delaying relapse in ANCA-associated vasculitis (AAV) patients in remission, a Phase 3 clinical trial shows. However, a subgroup of patients who receive Rituxan (rituximab) as an induction treatment may benefit from belimumab maintenance, the…
In light of upcoming Phase 3 data, ChemoCentryx and Vifor Fresenius Medical Care Renal Pharma have decided not to pursue the conditional approval of avacopan in Europe for patients with ANCA-associated vasculitis. Instead, the companies plan to submit a full (unconditional) marketing approval application for the therapy in 2020 with…
Anti-neutrophil cytoplasmic autoantibody (ANCA) testing is useful for the early diagnosis of ANCA-associated vasculitis (AAV) in patients with central nervous system symptoms, allowing for timely treatment and a better prognosis, but a positive ANCA test could also mean several things besides vasculitis, a review study reports. About 5-15 percent of…
An analysis of the gene expression and gene pathways in different tissues affected by ANCA vasculitis has provided new information about the mechanisms involved in disease progression, a study says. The study, “Gene Expression Pathways across Multiple Tissues in Antineutrophil Cytoplasmic Antibody-associated Vasculitis Reveal Core Pathways…
Impaired renal function, the presence of ANCA antibodies against the MPO protein, and a score of 2 or higher in the five-factor score — which measures prognosis at diagnosis in ANCA-associated vasculitis (AAV) — are all potential predictors of poor renal outcomes in children with AAV, a study has found.
The immunosuppressant agent mycophenolate mofetil (MMF) is similar to standard cyclophosphamide at inducing remission in ANCA-associated vasculitis (AAV) patients, but it seems to be associated with a higher relapse rate, a clinical trial shows. The findings suggest that MMF is a potential alternative to cyclophosphamide, particularly in patients…
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