News

Low blood levels of C3 protein at time of diagnosis are associated with poorer outcomes in patients with anti-neutrophil cytoplasmic antibodies (ANCA)-associated vasculitis (AAV), a Croatian study shows. The study, “Serum C3 complement levels in ANCA associated vasculitis at diagnosis is a predictor of patient and renal…

RIPK1 inhibitors may one day treat patients with antineutrophil cytoplasmic antibody (ANCA)-induced vasculitis (AAV), a new study finds. The study, “Necroptosis controls NET generation and mediates complement activation, endothelial damage, and autoimmune vasculitis,” appeared in the journal PNAS. AAV is characterized by circulating antibodies (ANCA) against…

Dr. Ronald J. Falk, chair of the department of medicine at the University of North Carolina and director of the UNC Kidney Center, was honored with the John P. Peters Award by the recent American Society of Nephrology (ASN) for his outstanding contributions to both research and patient care in kidney disease.

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…

Classifying patients with anti-neutrophil cytoplasmic antibodies (ANCA)-associated vasculitis (AAV) based on organ involvement at the time patients are first examined has stronger correlation with survival rates, than the customary classification based on disease phenotype, a study has found. The study, “Long-term patient survival in a Swedish population-based cohort…

Members of the U.K.-based European Vasculitis Society (EUVAS) have accepted recently published recommendations for the management of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV). However, EUVAS clinicians don’t necessarily agree with the recommendations’ suggestion to use Rituxan (rituximab) to treat patients who have eosinophilic granulomatosis with polyangiitis (EGPA). The study, “Validation…

Methotrexate is an effective and safe option for remission maintenance therapy in patients with anti-neutrophil cytoplasm antibody (ANCA)-associated vasculitis (AAV), a study has found. The study, “Methotrexate versus cyclophosphamide for remission maintenance in ANCA-associated vasculitis: A randomised trial,” was published in the journal Plos One. AAV…

Long-term maintenance therapy with azathioprine (AZA) does not improve relapse rates in patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), according to researchers. The study, “Long term azathioprine maintenance therapy in ANCA-associated vasculitis: combined results of long-term follow-up data,” was published in the journal…

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…