Two Phase 3 clinical trials will evaluate the safety and effectiveness of Roche’s investigational, self-injectable therapy crovalimab (RG6107) in children, adolescents, and adults with atypical hemolytic uremic syndrome (aHUS). The first trial, called COMMUTE-a (NCT04861259), already is recruiting aHUS patients, ages 12 and older, at sites in the…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
A newly identified mutation in the C3 gene that causes severe, life-threatening atypical hemolytic uremic syndrome (aHUS) was found in a baby boy in Israel, scientists report. While C3 mutations leading to overactivation of complement protein C3 are associated with aHUS, they are usually found in only one of…
Soliris (eculizumab) may help to control microangiopathy — a disease affecting small vessels — and restore kidney function in newborns with atypical hemolytic uremic syndrome (aHUS), according to a recent case report. The report, “Neonatal atypical hemolytic uremic syndrome in the eculizumab era,” was published…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
Researchers have reported a rare case of atypical hemolytic uremic syndrome (aHUS) in a 21-year-old Swiss-Indonesian man with lipoprotein glomerulopathy (LPG), an inherited kidney disease associated with fat accumulation in the organ’s small blood vessels. The findings suggest that the later development of aHUS, which also affects the kidney’s…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
A 6-month-old girl who was diagnosed with cystic fibrosis (CF) went into kidney failure due to atypical hemolytic uremic syndrome (aHUS), according to scientists who worked on her case report. Her kidney function was completely restored following hemodialysis, plasma exchange therapy — a form of treatment that replaces…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
Soliris (eculizumab) is effective for treating relapses in patients with atypical hemolytic uremic syndrome, even in the presence of severe kidney disease, according to a case report. The case study describes a 29-year-old woman with aHUS who experienced a relapse complicated by severe kidney disease, after recovering from…
Get regular updates to your inbox.