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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…
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…
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…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
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 National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
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…
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…
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…
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…
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