News

Soliris (eculizumab) maintenance treatment given every three weeks — instead of the standard two weeks — was sufficient to achieve therapeutic blood levels and prevent disease relapse for at least four years in a 2-year-old girl with atypical hemolytic uremic syndrome (aHUS), a case study shows. Soliris’ optimized regimen…

Difficulties in diagnosing atypical hemolytic uremic (aHUS) syndrome in the absence of family history are illustrated in the story of a girl in Sri Lanka who, after multiple diagnoses, was successfully treated for aHUS by long-term plasma exchange, a case study reported. …

Pregnancy-associated atypical hemolytic uremic syndrome (aHUS) usually occurs postpartum, after a pregnancy complication — such as preeclampsia, severe bleeding, and fetal death — and is treated effectively with Soliris (eculizumab), according to a review study. The data highlighted the previously unexplored variety of clinical scenarios in which…

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…

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…

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…