News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

Patients with granulomatosis with polyangiitis, a subtype of ANCA-associated vasculitis (AAV), have a higher rate of hospitalizations than the general population, a U.S. study reports. Their hospitalizations were also linked to higher morbidity, mortality, and costs. The study, “Inpatient epidemiology and economic burden of granulomatosis with polyangiitis: a…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

Lung involvement increases the risk for both induction therapy resistance and disease relapse in ANCA-associated vasculitis (AAV) patients with autoantibodies against myeloperoxidase, a Chinese study suggests. High levels of creatinine was also found to be a risk factor for treatment resistance. These findings, while needing further confirmation,…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

In people with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), central nervous system involvement is associated with more active disease and peripheral nervous system involvement, a recent Chinese study suggests. The study, “Central nervous system involvement in patients with antineutrophil cytoplasmic antibody–associated vasculitis: a study of 29…

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar.  William Whitman…

A combination of both blood tests and biopsy (tissue) analysis was better at predicting end-stage renal disease in people with anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV) compared to tissue analysis alone, a study has found.  The scientists recommended using the AAV renal…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Plasma exchange does not improve survival or prevent serious kidney disease in people with ANCA vasculitis, a study found. In addition, lower doses of glucocorticoids — steroid hormones that can ease inflammation — seem to be as effective as standard doses in terms of survival and kidney disease,…