News

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…

ChemoCentryx remains on track to report topline results later this year from its ongoing Phase 3 clinical trial assessing the safety and efficacy of oral avacopan (CCX168) in patients with ANCA-associated vasculitis (AAV), the company announced. Avacopan is an investigational inhibitor of the complement C5a receptor,…

In patients with MPO-positive ANCA-associated vasculitis, the excess inflammation causes cerebral small vessel disease even before the disease is diagnosed, and the risk for cerebral disease may remain high even after immunosuppressive treatment, researchers say. The study with that finding, “Occurrence of cerebral small vessel disease…

A large proportion of patients with ANCA-associated vasculitis discontinue treatment while in remission, but they appear to have fewer relapses than those who never stopped treatment, a study has found. This suggests that, as long as patients and physicians remain in touch to identify relapses, patients in remission may…

Tumors in the thymus can lead to a malfunction of the immune system, and cause the appearance of ANCA-associated vasculitis, a case report suggests. However, the association is rare, and more cases are needed to establish a line of treatment. The study, “Microscopic polyangiitis associated with thymic tumor:…

Lupus nephritis patients positive for ANCA antibodies show mixed symptoms of systemic lupus erythematosus and ANCA-associated vasculitis, culminating in aggressive kidney disease that does not respond well to remission-inducing therapies, a case report suggests. The study, “Rapid progression to end‐stage renal disease in a young female with mixed…

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

A new test that recognizes anti-neutrophil cytoplasmic autoantibodies (ANCAs) targeting proteins other than myeloperoxidase (MPO) and proteinase-3 (PR-3) could improve the diagnosis and classification of ANCA-associated vasculitis, a study suggests. The study, “ANCA Beyond MPO & PR-3: the Clinical Utility of an Expanded ANCA Profile,” was recently presented at…

The levels of two molecules in the blood — eosinophil cationic protein (ECP) and interleukin-5 (IL-5) — reflect eosinophil activation in patients with eosinophilic granulomatosis with polyangiitis (EGPA), a study shows. As a result, these molecules may be used as markers to determine disease activity in EGPA patients and distinguish…

Genentech‘s Rituxan (rituximab) is safe and effective to induce and maintain remission in patients with refractory or relapsing granulomatous manifestations (inflamed tissue) due to granulomatosis with polyangiitis (GPA), according to a new study. The findings were presented at the recent 19th International Vasculitis and ANCA Workshop in Philadelphia, Pennsylvania,…