News

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…

The Foundation for Sarcoidosis Research (FSR) announced its newest initiative this month, the African American Women & Sarcoidosis National Campaign, to bring awareness to how this group is disproportionately affected by the disease and to educate people living with the disorder. African American women are three times more…

White women with sarcoidosis and multiorgan involvement may be at a higher risk of hypothyroidism, or low thyroid gland activity, than other people with the overactive immune system disorder, according to a study of more than 3,800 patients in the U.S. In fact, hypothyroidism — which is significantly…

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…

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.

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…

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.

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

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…