News

Sangamo Therapeutics is preparing to launch a Phase 1/2 clinical study in the United Kingdom to explore the potential of its cell-based CAR-Treg therapy — TX200 — in preventing immune-mediated rejection of transplanted kidneys in patients with end-stage renal disease. The U.K.’s Medicines Healthcare Products Regulatory…

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

Severe and life-threatening hypertension is common in people with atypical hemolytic uremic syndrome (aHUS), and Soliris (eculizumab) is a more effective than plasmapheresis in treating these patients, a Spanish study finds. The study, “Severe and malignant hypertension are common in primary atypical hemolytic uremic syndrome,”…

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…

The U.S. Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz) for the treatment of adults and children ages one month and older with atypical hemolytic uremic syndrome (aHUS), Alexion Pharmaceuticals announced. This is the first time the medication, previously approved to…

Treatment of atypical hemolytic uremic syndrome (aHUS) with Soliris (eculizumab) should be maintained even after clinical deterioration, a case report suggests. This is the first report of suboptimal response to Soliris in a patient with aHUS associated with mutations in both the CFI and THBD genes, the…

Treatment with Soliris (eculizumab) led to successful resolution of atypical hemolytic uremic syndrome (aHUS) that was triggered by inflammatory bowel disease (IBD), a study of two cases has found. The study, “Successful use of eculizumab to treat atypical hemolytic uremic syndrome in patients with inflammatory bowel…

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…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told BioNews Services, publisher…

Two diseases often difficult for doctors to distinguish between — atypical hemolytic uremic syndrome (aHUS) and thrombotic thrombocytopenic purpura (TTP) — are both marked by complement system activation and severe deficiency of the ADAMTS13 enzyme, researchers report. These disorders are known to share symptoms, but until now were largely…