News

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

DNA Sequencing May Help ID Tuberculosis Versus Sarcoidosis

A DNA sequencing technology called next-generation sequencing (NGS) may be used to detect tuberculosis (TB) rapidly and accurately — which could help in distinguishing it from sarcoidosis, a study suggests. While tuberculosis and sarcoidosis share many symptoms, the therapies for the two diseases differ greatly, according to researchers.

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…