Guidelines for using rituximab as a maintenance therapy in adults with ANCA-associated vasculitis (AAV) were recently published that, in addition to helping physicians with treatment approaches, highlight areas where more research is needed. “Rituximab for maintenance of remission in ANCA-associated vasculitis: expert consensus guidelines,” was…
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.
Continuing maintenance treatment with rituximab for long periods increases the likelihood that people with ANCA-associated vasculitis (AAV) remain in remission, without increasing the incidence or severity of adverse events, findings from a Phase 3 trial show. The study, “Long-Term Rituximab Use to Maintain Remission of…
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…
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