News

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

Changes in autoantibodies that target the protein PR3 in people with ANCA-associated vasculitis (AAV) may help predict sustained remission following treatment, and eventual disease relapses, a study found. The research also shows that the ALBIA method, which is becoming widely…

Avacopan is superior to prednisone at improving kidney health in people with ANCA-associated vasculitis (AAV) and poor kidney function, and in extending time until a disease relapse, new data from a Phase 3 trial show. These findings were recently shared at ACR Convergence 2020, the virtual annual meeting of…

Measuring the level of C-reactive protein monomer (mCRP) — a protein that increases in response to tissue damage and inflammation — may help identify those ANCA-associated vasculitis (AAV) patients who have cardiovascular disease, a recent study found.  The study, “The plasma level of…

The European Medicines Agency (EMA) has agreed to review Vifor Pharma and ChemoCentryx’s application requesting the approval of avacopan to treat granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA), the companies announced in a press release. GPA and MPA are two types of…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…

People with ANCA-associated vasculitis (AAV) have a similar incidence of COVID-19 as the general population, but the ongoing pandemic has significantly disrupted routine medical care for patients with this autoimmune disease, a study found. Some AAV patients had therapies postponed or had healthcare visits rescheduled; 16% had less…

Having persistently or recurrently high levels of ANCA antibodies is associated with a greater risk of relapse in people with ANCA-associated vasculitis (AAV), a recent study has found. In patients whose ANCA antibodies target the myeloperoxidase (MPO) protein, a recurrent ANCA pattern was also significantly associated with…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…