News

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

Certain Immune Cells May Help Assess AAV Disease Activity

A blood parameter called large unstained cell count or LUC – which measures the numbers of certain activated immune cells – appears to associate with higher ANCA-associated vasculitis (AAV) disease activity, a small study found. These results, when combined with the findings of previous, larger-scale studies, show that “LUC…

Nominations Open for 2022 Eurordis Black Pearl Awards

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

ChemoCentryx Amends Approval Application for Avacopan

ChemoCentryx has filed an amendment to its application seeking approval of avacopan, an investigational therapy for the treatment of ANCA-associated vasculitis (AAV). The filing addresses the issues raised by an advisory committee of independent medical experts during a recent meeting with the U.S. Food and Drug…

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

Abstract on How Avacopan Affects Kidney Function Makes Top 10 List

Treatment with ChemoCentryx’s investigational therapy avacopan can improve kidney function in people with ANCA-associated vasculitis to a greater extent than steroids, especially among individuals with advanced kidney disease, clinical trial data show. The data were presented at the annual meeting of the European Renal Association –…