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More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…

About a fifth of ANCA-associated vasculitis patients have an eye disorder known as inflammatory ocular disease, a Mayo Clinic study shows. The research, published in the journal Rheumatology, was titled “Clinical characteristics of inflammatory ocular disease in anti-neutrophil cytoplasmic antibody associated vasculitis: a retrospective cohort study.” ANCA-associated vasculitis, or AAV, affects…

Although certain immune cells are present at lower levels in patients with anti-neutrophil cytoplasm antibody (ANCA)-associated vasculitis (AAV), it is due to non-specific renal injury rather than the disease itself, study shows. The study, “Alterations in circulating lymphoid cell populations in systemic small vessel vasculitis are non-specific…

Different types of ANCA-associated vasculitis occur in different ethnic groups, a study reports. The research, “Global ethnic and geographic differences in the clinical presentations of anti-neutrophil cytoplasm antibody􏰀associated vasculitis,” was published in the journal Rheumatology. Anti-neutrophil cytoplasm antibodies-associated vasculitis, or AAV, refers to three…

The protein TIMP1 is a promising biomarker of ANCA-associated vasculitis activity, according to a Japanese study that also identified markers of the disease’s damage to the kidneys and lungs. Altogether, the researchers identified nine proteins that could be suitable biomarkers. Researchers published their work in the journal Arthritis Research…

Lower levels of key immune system regulators in the kidneys of  ANCA vasculitis patients may indicate more severe renal injury, according to a new study from China. The study, “Complement regulatory proteins in kidneys of patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis,” appeared in the journal…

A signaling molecule produced by a type of immune T-cell plays a key role in the development of the kidney inflammation glomerulonephritis, an Australian study reports. The molecule is interleukin-17, or IL-17. The cell that generates it is the gamma-delta (γδ) type of T-cell. And the kidney condition is myeloperoxidase (MPO) anti-neutrophil…

Certain types of patients with ANCA-associated vasculitis do not require maintenance therapy if treated with a specific Rituxan (rituximab) approach, according to researchers at the University of Turin in Italy who followed patients for up to 11 years. The study, albeit small, might clear up some confusion about the…

Dutch scientists have identified different genes in Staphylococcus aureus — a kind of bacteria carried in the nostrils — in either patients with PR3-ANCA vasculitis or with MPO-ANCA-positive vasculitis. The results suggest that the genes they found are relevant in causing and prolonging the disease. The study, “Genetic loci…