PANTHERx, Rare Disease Pharmacy, to Distribute Tavneos in US

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…

I’ve been writing my column, “Peaceful Chaos,” about living with granulomatosis with polyangiitis (GPA) since June. I’ve written about my medications, doctor appointments, and the daily struggles of being chronically ill. I’ve discussed the psychological effect this disease has had on me and on others, and what…

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…

People are social creatures who thrive when nurtured, and when our achievements — such as weddings, graduations, promotions, and new babies — are celebrated. But when challenges arise, a supportive foundation can prove even more valuable. After I was diagnosed with granulomatosis…

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…

Males are not at a greater risk than females of experiencing kidney failure or death due to ANCA-associated vasculitis (AAV), regardless of the kind of kidney damage they have, a study found. These new findings counter the results of a 2018 study that had reported an increased risk of…

The year I turned 30, I experienced a vague sense of panic. It seemed like the age at which the fundamental aspects of life — career, family, hobbies — coalesce into permanence. I had recently moved across the country to resettle in a new city that I enjoyed. My work…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

The Japanese Ministry of Health, Labor, and Welfare (MHLW) has approved Tavneos (avacopan) for the treatment of microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA). The approval of this first-in-class therapy — for the two main forms of ANCA-associated vasculitis (AAV) — was granted to Kissei Pharmaceuticals, which…