The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…
News
On the outside, Khloe Sudol seems like a normal 11-year-old. The quiet, reserved girl is full of energy, filling her time with horseback riding, swimming, and school work. But on the inside, her kidneys have failed as a result of the ultra-rare disease atypical hemolytic uremic syndrome (aHUS), which…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
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…
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