Get regular updates to your inbox.
A Patient Speakers’ Bureau is opening at the Foundation for Sarcoidosis Research (FSR) to advocate on behalf of people with this rare disease of the immune system and bring greater awareness to it. Fourteen trained and experienced patient advocates will use their personal stories to help others in understanding…
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…
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 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…
The use of Acthar Gel (repository corticotropin injection, or RCI) was among medications noted by both European and U.S. panels of experts, identified as a possible treatment of pulmonary sarcoidosis when other therapies fail or are not tolerated. Notably, the therapy is currently approved to treat active sarcoidosis only…
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…
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…
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…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
Get regular updates to your inbox.