Author Archives: Patricia Inacio PhD

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…

Successful kidney transplant for atypical hemolytic uremic syndrome (aHUS) is aided by treatment with Soliris (eculizumab), a small Turkish study has found. The study “Renal Transplantation in Patients With Atypical Hemolytic Uremic Syndrome: A Single Center Experience” was published in the journal Transplantation Proceedings. Hemolytic uremic syndrome (HUS) is…

Therapies against the complement C5 protein, the target of Soliris (eculizumab), may be more effective than targeting proteins downstream of the C5 protein in the complement system signaling cascade in atypical hemolytic uremic syndrome (aHUS), a new mouse study shows. The study, “Differential contribution of C5aR…

New research shows how an antibody was able to enhance the function of a member of the complement system, called complement factor H (FH), responsible for keeping this system in check. Augmenting FH with the newly discovered antibody reduced red blood cell burst in blood samples of atypical hemolytic uremic…