News

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…

New Institute Aims to Leave No Rare Disease Patient Behind

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…

Group Focuses on Rare Disease Clinical Trial Participation

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…

Boosting Cell Stress Response May Be Therapeutic Approach in aHUS

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…

Crohn’s Disease May Lessen Soliris’ Effectiveness in aHUS Patients

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…