News

The case of a woman with unusual symptoms for atypical hemolytic uremic syndrome (aHUS) — including normal hemoglobin levels and mildly low platelet counts — highlights the need for better and more clear diagnostic criteria for this condition, according to researchers. The goal is to ensure that patients can…

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…

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…

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…

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…

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…

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…

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…

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…