Author Archives: Patricia Inacio PhD

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…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Treatment with Soliris (eculizumab) successfully prevented the recurrence of atypical hemolytic uremic syndrome (aHUS) in a 64-year-old woman who had received a kidney transplant, without compromising her recovery from COVID-19, a case study reports. The study, “COVID-19 in a kidney transplant recipient treated with eculizumab for…

Adults with atypical hemolytic uremic syndrome (aHUS) continue to show improvements in kidney function, blood parameters, and quality of life after receiving Ultomiris (ravulizumab) for a median of 1.5 years, new analysis of clinical trial data suggests. The findings were reported in the study “Long-Term Efficacy and…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Treatment with Soliris (eculizumab) during pregnancy may help prevent subsequent relapses of pregnancy-associated atypical hemolytic uremic syndrome (aHUS), according to a case report. The report, “Eculizumab Maintenance and the Prevention of Atypical Hemolytic Uremic Syndrome Relapse During Pregnancy: A Case Report,” was published in the…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

A rare case of a 20-year-old male with atypical hemolytic uremic syndrome (aHUS) showing damage to both eyes that culminated in impaired vision highlights the importance of prompt diagnosis and treatment to ensure better outcomes, a report says. The study, “Bilateral proliferative retinopathy and ischemic optic neuropathy in a…

Treatment with Soliris (eculizumab) in pregnant women with atypical hemolytic uremic syndrome (aHUS) may not increase the risk of miscarriage, according to the analysis of a small group of patients enrolled in the Global aHUS Registry. Marie Scully, MD, of University College London, presented the results of the analysis at the…