News

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

A novel index that computes five simple laboratory results at the time of diagnosis may improve predictions of all-cause mortality among patients with ANCA-associated vasculitis (AAV), a South Korean study reports. These lab results include the levels of immune neutrophils, lymphocytes, and monocytes, and of proteins C-reactive protein and…

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

Rituximab is safe and effective for inducing remission in two types of ANCA-associated vasculitis in children and adolescents: granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), according to data from a Phase 2a trial. After one and a half years on the trial, all patients achieved remission, and safety…

A reduced dosage of rituximab maintenance therapy may be effective at preventing relapses among people with ANCA-associated vasculitis (AAV), a new study reports. Specifically, the researchers said 500 mg infusions every six months “appear to be an effective and safe option” for maintaining AAV remission. “This study provides real-world…

Symptoms differ among people with the rare disorder eosinophilic granulomatosis with polyangiitis (EGPA), depending on whether they test positive or negative for antineutrophil cytoplasmic antibodies (ANCAs). A recent case study revealed that ANCA-positive patients tended to have more blisters, body-wide inflammatory symptoms, and to receive higher doses of oral…

A two-dose rituximab regimen is as safe and works as well as the standard four doses in inducing remission in adults with severe granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), according to a review study. While these findings support the current use of either regimen in clinical practice…

The American College of Rheumatology and the Vasculitis Foundation have published new guidelines to improve the treatment and management of systemic vasculitis, a group of inflammatory disorders that includes ANCA-associated vasculitis (AAV). The rarity of these disorders can make them challenging to diagnose and treat. The new…

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

A blood parameter called large unstained cell count or LUC – which measures the numbers of certain activated immune cells – appears to associate with higher ANCA-associated vasculitis (AAV) disease activity, a small study found. These results, when combined with the findings of previous, larger-scale studies, show that “LUC…