News

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…

A positive anti-proteinase 3 (PR3) ANCA test, lower serum creatinine levels, and cardiovascular involvement at diagnosis appear to raise the risk of relapse in people with ANCA-associated vasculitis (AAV), a review study reports. When combined, these factors may predict an individual’s risk of relapse within five years after diagnosis,…

Treatment with tofacitinib is well tolerated and effectively induces remission in people with ANCA-associated vasculitis (AAV), while enabling patients to lower their glucocorticoid doses, a small pilot study suggests. These results indicate that tofacitinib may represent a treatment option for active AAV. However, a larger and randomized controlled trial is needed…

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…