News

Mycophenolate mofetil (MMF) may be a safe and effective alternative to the standard induction therapy cyclophosphamide in certain ANCA-associated vasculitis (AAV) patients, a review study reports. Based on the data, researchers recommend that MMF be used in patients with autoantibodies against myeloperoxidase (MPO), non-life-threatening disease, and those…

InflaRx announced it has fully enrolled the first part of its Phase 2 trial assessing the safety and efficacy of IFX-1 in people with ANCA-associated vasculitis (AAV) in Europe. The company also completed a blinded interim analysis into the potential impact of COVID-19 on this…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

ChemoCentryx is planning to file a request with the U.S. Food and Drug Administration (FDA) by mid-year seeking approval of avacopan (CCX168) as a treatment for people with ANCA-associated vasculitis (AAV), the company said. Its new drug application will be supported by results of the Phase 3 ADVOCATE trial (…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

A common blood pressure medication called hydralazine may cause ANCA-associated vasculitis (AAV), with a severe impact on both lungs and kidneys in rare instances, a case report shows. The medication has been widely associated with AAV, but reports of severe lung and kidney injury are uncommon. In such…

A rare case of ANCA-associated vasculitis (AAV) causing thickening of the dura mater — a protective membrane of the central nervous system (CNS) — was found in a person previously treated for ANCA-associated interstitial lung disease. The case report was detailed in the study, “A…

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…