News

A new classification system, based on the underlying cause of disease, may be necessary to better understand ANCA-associated vasculitis (AAV) and other disorders characterized by inflammation in the blood vessels. That’s according to a team led by scientists at Aarhus University, in Denmark, who published their idea for the…

After an initial biopsy shows signs of active ANCA-associated vasculitis (AAV) affecting the kidneys, repeat biopsies are better at determining active disease than standard measures of kidney function, a new observational study reports. Such repeat kidney biopsies may help determine treatment response and personalize care plans for people with…

Kidney disease is less common among people with eosinophilic granulomatosis with polyangiitis (EGPA), but some patients may experience severe kidney damage and require management by specialists, a case report shows. The study, “Renal Involvement in Eosinophilic Granulomatosis With Polyangiitis,” was published in the journal Kidney International Reports.

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…

A woman of childbearing years developed ANCA-associated vasculitis (AAV) after taking the thyroid medication methimazole to treat her Graves’ disease, a case report shows. The patient experienced signs of respiratory and kidney failure secondary to AAV, but her symptoms improved after discontinuing the medication and starting immunosuppressive medications. Four…

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…

Tavneos (avacopan), a recently approved add-on oral therapy for severe, active ANCA-associated vasculitis (AAV), will be distributed in the U.S. by PANTHERx, the country’s leading rare disease pharmacy. The announcement follows an agreement with ChemoCentryx, Tavneos’ developer, which selected the pharmacy as a limited distribution partner. “PANTHERx is proud…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

The U.S. Food and Drug Administration (FDA) has approved Tavneos (avacopan) as an add-on therapy for people with severe active microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA), the two most common forms of ANCA-associated vasculitis. The approval comes after an advisory committee narrowly supported avacopan’s approval…