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…

Trial of ATYR1923 in Pulmonary Sarcoidosis Patients Nears Finish

A Phase 1/2 clinical trial testing ATYR1923 in people with pulmonary sarcoidosis has now finished all required patient visits. Results from this proof-of-concept study are expected this September. “We are pleased to complete the last patient visit in this important study, which represents a significant milestone for aTyr, our ATYR1923 clinical program and…

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…

Neurofilament Protein Is Promising Neurosarcoidosis Biomarker

A protein called neurofilament light chain (NFL), which supports nerve fibers, is elevated in adults with neurosarcoidosis — and appears to increase with more extensive brain inflammation — a study demonstrated. These findings support the neurofilament protein as a biomarker for disease activity in neurosarcoidosis, a form of…

Xentria Readies First Trial of XTMAB-16 for Sarcoidosis

The U.S. Food and Drug Administration (FDA) has approved beginning clinical trials testing XTMAB-16, Xentria’s investigational TNF-alpha inhibitor for the treatment of sarcoidosis. The first, a Phase 1 trial evaluating the therapy in healthy volunteers, is expected to start in July at the Parexel Baltimore Early Phase…

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…