Alexion Pharmaceuticals now holds exclusive rights to Dicerna’s potential RNA interference (RNAi) therapies developed against four targets of the complement system, which plays a role in many diseases, including atypical hemolytic uremic syndrome (aHUS). aHUS results from an abnormal activation of the complement system, a…
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Plasma exchange (PEX) therapy given before and after kidney transplants reduces the risk of disease relapse and transplant failure in people with atypical hemolytic uremic syndrome (aHUS), according to an Austrian study. The work also suggested that while treatment with Soliris (eculizumab) may not be necessary for all…
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
The activity of a molecule called ADAMTS13 can predict the outcome of patients with with atypical hemolytic uremic syndrome (aHUS) and their response to plasma exchange therapy (PEX), as well as guide treatment decisions, a Korean study suggests. The study, “…
A newly developed blood test is able to rapidly identify complement system dysregulation in people with conditions related to thrombotic microangiopathies (TMAs) — including atypical hemolytic uremic syndrome (aHUS) — as well as monitor aHUS patients’ responses to Soliris (eculizumab) treatment, researchers said. The test, a…
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Sangamo Preparing to Launch UK Clinical Trial Testing TX200 Cell Therapy in End-stage Renal Disease
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…
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Severe Hypertension Common in aHUS Patients and Soliris Effective at Treating Them, Study Finds
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…
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