News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

Inflammatory vaginitis — inflammation of the vagina — may develop in women receiving long-term treatment with rituximab for ANCA-associated vasculitis (AAV) or other autoimmune diseases, a U.S. study says. The findings suggest that “women treated with rituximab should be screened for symptoms such as vaginal discharge and vaginal pain,” its…

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…

Mycophenolate mofetil (MMF) is well-tolerated and can be used to induce remission in people with eosinophilic granulomatosis with polyangiitis (EGPA), a type of ANCA-associated vasculitis (AAV), a real-world study shows. Two-thirds of patients with newly diagnosed or relapsing EGPA achieved remission, or had no symptoms appearing for a time,…

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

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…