News

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…

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…

Severe colon infection caused by the bacteria Clostridioides difficile triggered a rare case of atypical hemolytic uremic syndrome (aHUS) in a healthy young woman, a case study reported. She responded quickly to treatment with Soliris (eculizumab), its scientists noted, leading them to suggest this bacterial infection can be…

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…