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,…
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
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…
ANCA-associated vasculitis patients in Korea have a high prevalence of thyroid dysfunction, with the condition affecting mainly those who are older, have ANCA antibodies, or renal involvement at diagnosis, a study has found. The study, “Thyroid Dysfunction in Patients with Antineutrophil Cytoplasmic Antibody−associated Vasculitis: A Monocentric Retrospective Study,”…
Treatment using plasma exchange can help ANCA-associated vasculitis patients with severe renal damage become independent of dialysis in just a few weeks, according to a study. The study, “Plasma exchange is highly effective for ANCA-associated vasculitis patients with rapidly progressive glomerulonephritis who have advanced to dialysis…
The Birmingham Vasculitis Activity Score (BVAS) and the Five Factors Score (FFS) are both useful tools for predicting the prognosis of ANCA-associated vasculitis, and for selecting the best first-line therapy, a study says. Researchers recommended using both for best patient care. The study, “Comparison of…
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