News

RARE-X, Global Genes to Help Collect Rare Disease Patient Data

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…

Global Genes, Diversity Coalition Team Up to Advance Health Equity

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

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…

aHUS Found in Boy With Rare, Severe Immune Deficiency Disorder

Atypical hemolytic uremic syndrome (aHUS) can co-exist with severe combined immunodeficiency (SCID), a rare genetic disorder characterized by a weakened immune system, a case report noted. Prompt diagnosis is crucial to improving patient outcomes, its researchers wrote, especially as the two diseases “are considered to exacerbate each other.” The…

‘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…

Discontinuing Soliris Is Safe for Certain aHUS Patients

Treatment with Soliris (eculizumab) may be discontinued with close monitoring in people with atypical hemolytic uremic syndrome (aHUS) whose disease is in remission for at least six months — providing their kidney function remains stable — or in those who have developed end-stage kidney disease, a small study…

Supporters Gear Up for aHUS Awareness Day on Sept 24

From participating in an organ donor registration drive to sharing a community video project, supporters globally are poised to mark the 7th annual aHUS Awareness Day on Sept. 24. The event seeks to heighten visibility around atypical hemolytic uremic syndrome (aHUS), highlight the latest research, and underscore the needs…