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…
Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…
Infection with SARS-CoV-2, the virus that causes COVID-19, may trigger first clinical symptoms or relapse of atypical hemolytic uremic syndrome (aHUS) in people carrying mutations in complement genes, a case report suggests. The report, “Atypical HUS triggered by infection with SARS-CoV2,” was published in the journal…
More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…
Family members of people with atypical hemolytic uremic syndrome (aHUS) who carry mutations in complement genes may be at a lower risk of developing this disease than previously reported, according to an Italian study. The single-center study estimated the risk to be at 20% — much lower than the 50%…
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…
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…
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