News

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…

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…

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…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…