A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
Neutrophils from patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) are better at activating the alternative complement system than neutrophils from healthy people, a study has found. This research emphasizes the role of complement in AAV disease development, and suggests the therapeutic potential of its inhibition. The study, titled…
Treatment with Rituxan (rituximab) had some efficacy in eosinophilic granulomatosis with polyangiitis patients, leading to a reduction in glucocorticoid requirement, but asthma and ear, nose and throat (ENT) relapse rates remained high despite continued treatment, a study found. The study, “Efficacy and safety of rituximab in the…
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
In a new study comparing Rituxan (rituximab) and azathioprine for the treatment of ANCA-associated vasculitis (AAV), rituximab was found to be a cost-effective option, even though it is the more expensive treatment. The study, “Cost-effectiveness of rituximab versus azathioprine for maintenance treatment in antineutrophil…
People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
The neutrophils of patients with granulomatosis with polyangiitis (GPA) have altered levels of proteins involved in neutrophil clearance, which may contribute to the blood vessel inflammation and autoimmunity marking this disorder — especially in patients with kidney damage — a study has found. Harnessing these altered pathways could lead…
The U.S. Food and Drug Administration (FDA) has granted Priority Review to Rituxan (rituximab) in combination with glucocorticoids for the treatment of children, 2 or older, with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). If Genentech‘s Biologics License Application (sBLA) for Rituxan is approved, this…
People who live in the U.S. and have eosinophilic granulomatosis with polyangiitis (EGPA) now can be treated with Nucala (mepolizumab) once every four weeks through at-home injections. The U.S. Food and Drug Administration (FDA) recently approved an autoinjector and a pre-filled safety syringe as new methods to dose…
People with ANCA-associated vasculitis with high levels of myeloperoxidase (MPO) antibodies at diagnosis are more likely to have poor kidney function and lower 2-year renal survival, a study says. The study, “High anti-neutrophil cytoplasmic antibody titers are associated with the requirement of permanent dialysis in patients with myeloperoxidase-ANCA-associated…
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