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Ultomiris (ravulizumab) can safely and effectively treat women with atypical hemolytic uremic syndrome (aHUS) whose symptoms are triggered by pregnancy, a Phase 3 clinical trial analysis reveals.  The study, “Efficacy and safety of the long-acting…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

Ultomiris (ravulizumab-cwvz) is safe and effective in children with atypical hemolytic uremic syndrome (aHUS), data from a Phase 3 clinical trial show. These findings were in the study “The long-acting C5 inhibitor, ravulizumab, is effective and safe in pediatric patients with atypical hemolytic uremic syndrome naïve to…

COVID-19 was identified as the trigger for atypical hemolytic uremic syndrome (aHUS) in two adults, but the patients — one man and one woman — responded well to treatment with Soliris (eculizumab), according to a recent study. The study, “Case series: coronavirus disease 2019 infection as a…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

For Rare Disease Day on Feb. 28, aHUS Alliance Global Action is inviting people worldwide to take part in a video/slideshow project aimed at raising awareness of atypical hemolytic uremic syndrome (aHUS) and the needs of its community. To participate, patients and caregivers are asked to send a…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…