News

aTyr Pharma has announced a collaboration with The Ohio State University (OSU) to conduct cell culture studies seeking to identify potential biomarkers that can predict ATYR1923’s therapeutic effectiveness in people with pulmonary sarcoidosis. The research, which extends previous proof-of-concept findings, will delineate the pathways involved in sarcoid granuloma formation…

The U.S. Food and Drug Administration has granted orphan drug designation to RLF-100 (aviptadil), Relief Therapeutics’ investigational inhaled therapy for sarcoidosis. Such status is given to treatment candidates with the potential to be safe and effective in rare diseases — defined in the U.S. as those affecting fewer…

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…

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…

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…