News

ATYR1923 Found Safe, Effective for Pulmonary Sarcoidosis Patients

aTyr Pharma’s experimental therapy ATYR1923 safely and effectively improves lung function and reduces symptoms in adults with pulmonary sarcoidosis, while lowering the need for oral corticosteroids, top-line data from a Phase 1b/2a clinical trial show. Notably, while all three tested therapy doses were well-tolerated, greater clinically meaningful…

Group Focuses on Rare Disease Clinical Trial Participation

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

Phase 2 Trial of Inhaled Therapy Aviptadil OK’d in Germany

A Phase 2 clinical trial investigating the inhaled sarcoidosis therapy aviptadil — also known as RLF-100, and designed to block inflammation in the lungs — has been cleared to start in Germany. The clearance by the German medical regulatory authority Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM) was given to…

Researchers Seek Biomarkers to Predict ATYR1923’s Effectiveness

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…

Register Now for Global Genes’ RARE Patient Advocacy Summit

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

Advocates Lobby US Congress During Virtual Rare Disease Week

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…