News

Thyroid Med for Graves’ Disease Triggered AAV

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…

RARE-X, Global Genes to Help Collect Rare Disease Patient Data

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…

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…

Global Genes, Diversity Coalition Team Up to Advance Health Equity

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…

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

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…

FDA Approves Tavneos as Add-on Therapy for Severe MPA, GPA

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…

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

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.

ChemoCentryx’s Tavneos Approved in Japan for MPA, GPA

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…

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

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…