The U.S. Food and Drug Administration (FDA) has granted Priority Review to the supplemental Biologics License Application (sBLA) for Alexion‘s Ultomiris (ravulizumab-cwvz) for the treatment of patients with atypical hemolytic uremic syndrome (aHUS). aHUSÂ is caused by the progressive destruction of red…
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
Treatment with plasma exchange (PEX) was safe, restored kidney function and led to no requirement of dialysis at three months in most children with atypical hemolytic uremic syndrome (aHUS), according to research from India. The study, “Membrane-filtration based plasma exchanges for atypical hemolytic uremic syndrome: Audit…
People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
A novel genetic variant of the CFH gene, which encodes an important protein from the immune complement system, was linked to the development of atypical hemolytic uremic syndrome (aHUS) in a German woman. Her case was reported in a study, “…
In some patients, atypical hemolytic uremic syndrome (aHUS) may be associated with heart disease, a case report study highlights. The case report study, “Atypical Hemolytic Uremic Syndrome Presenting as Acute Heart Failure — A Rare Presentation: Diagnosis Supported by Skin Biopsy,” was published in the Journal…
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…
Rapid drug desensitization may allow patients with atypical hemolytic uremic syndrome (aHUS) to continue treatment with Soliris (eculizumab), following a medicine-induced allergic reaction, a case report says. The study, “Eculizumab hypersensitivity and desensitization in a toddler with atypical hemolytic uremic syndrome,” was published in The…
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies(NCATS) at the…
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