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…
Soliris (eculizumab) was shown to effectively treat atypical hemolytic uremic syndrome (aHUS) triggered by COVID-19 in a patient who had undergone a heart transplant five years earlier, according to a recent case report study. “We present a case of [a] 32-year-old Hispanic male with a history of heart…
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…
Endothelial cells, or those that line blood vessels, are functionally abnormal in people with atypical hemolytic uremic syndrome (aHUS) associated with antibodies against complement factor H or CFH, according to a small study using patient-derived cells. Notably, these intrinsic defects were associated with reduced activation of the p38 MAPK…
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…
Relapses of Crohn’s disease, an inflammatory bowel condition, lessened the efficacy of Soliris (eculizumab) in an adolescent girl with atypical hemolytic uremic syndrome (aHUS), a case study reported. “Increased doses of [Soliris] may be necessary to maintain therapeutic blood levels of [Soliris] and full complement blockade, especially if…
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