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December 11, 2019December 11, 2019

Preventive Plasma Therapy Reduces Risk of Kidney Transplant Failure, Austrian Study Shows

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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 ... Read more

December 6, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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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 ... Read more

December 4, 2019December 4, 2019

Study Links ADAMTS13 Activity to Improved PEX Treatment Outcomes in aHUS Patients

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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 ... Read more

November 27, 2019November 27, 2019

New, Modified Blood Test May Help Identify Complement Dysregulation in aHUS, Other TMAs, Study Suggests

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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) ... Read more

November 20, 2019November 20, 2019

Sangamo Preparing to Launch UK Clinical Trial Testing TX200 Cell Therapy in End-stage Renal Disease

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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 ... Read more

November 13, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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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. ... Read more

November 5, 2019November 5, 2019

Severe Hypertension Common in aHUS Patients and Soliris Effective at Treating Them, Study Finds

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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. ... Read more

October 29, 2019October 29, 2019

Rare Eye Manifestations Linked to aHUS, Study Reports

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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 ... Read more

October 23, 2019October 23, 2019

FDA Approves Alexion’s Ultomiris for Treating aHUS in Children and Adults

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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), ... Read more

October 16, 2019October 16, 2019

Clinical Deterioration Should Not Dictate Soliris Withdrawal, Report Suggests

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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 ... Read more

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