Atypical Hemolytic Uremic Syndrome News and Research

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Genetic analysis reveals hidden causes of chronic kidney disease in adults

Chronic kidney disease (CKD) is extremely prevalent among adults, affecting over 800 million individuals worldwide.

3 Apr 2024

Study establishes the ideal immunosuppression regimen for pig-to-human kidney xenotransplantation

Currently used Food and Drug Administration-approved transplant drugs -; with the addition of an also already FDA-approved complement inhibitor -; are the optimal immunosuppression regimen for pig-to-human kidney transplants, according to a landmark discovery by University of Alabama at Birmingham investigators.

25 Jan 2024

Researchers identify a promising method to stop COVID-19 infection

While the world waits eagerly for a safe and effective vaccine to prevent infections from severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the virus behind the COVID-19 pandemic, researchers also are focusing on better understanding how SARS-CoV-2 attacks the body in the search for other means of stopping its devastating impact.

8 Oct 2020

New technique to detect common cause for diseases related to thrombotic microangiopathies

There is a group of diseases whose symptoms have a common origin: a lesion in the endothelial tissue that covers the inside of the blood vessels.

8 Nov 2019

FDA approves first drug for treatment of neuromyelitis optica spectrum disorder

The U.S. Food and Drug Administration today approved Soliris (eculizumab) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder in adult patients who are anti-aquaporin-4 antibody positive.

27 Jun 2019

Omeros’ OMS721 receives positive opinion from EMA's COMP for treating hematopoietic stem cell transplant

Omeros Corporation announced today that the European Medicines Agency's Committee for Orphan Medicinal Products issued a positive opinion recommending orphan drug designation of OMS721 for treatment in hematopoietic stem cell transplantation.

25 Jul 2018

TSRI scientists reveal how vision-loss treatment may leave the eyes, kidneys vulnerable to cellular damage

A common class of drugs for vision loss may actually add to the problem in some patients, according to new research co-led by scientists at The Scripps Research Institute (TSRI).

7 Dec 2016

Alexion initiates new global Uncommon Strength campaign to raise awareness of rare diseases

Alexion Pharmaceuticals, Inc. today announced the launch of Uncommon Strength, a global campaign to raise awareness of rare diseases through the celebration of the extraordinary resilience and inner strength of those impacted by these diseases.

15 Sep 2016

Scientists use X-rays to study how eculizumab prevents immune system from damaging kidney tissue

In collaboration with Alexion Pharmaceuticals, Inc., scientists from Aarhus University have used X-rays to understand how the therapeutic antibody eculizumab prevents our immune system from destroying red blood cells and damaging kidney tissue.

6 Jun 2016

New lab blood test may help identify HELLP syndrome in pregnant women

A laboratory blood test developed at Johns Hopkins for the diagnosis of a rare genetic red blood cell disorder also shows promise in identifying HELLP syndrome, a life-threatening high blood pressure condition affecting 1 percent of all pregnant women that causes hypertension along with end organ damage, researchers report in the May issue of the journal Experimental Hematology.

19 May 2016

Pabetalone (RVX-208) to be tested in proof-of-concept pilot study in complement mediated diseases

Resverlogix Corp. announced today the commencement of an Orphan Disease Program specific for Complement Mediated Diseases. New data generated by Resverlogix has demonstrated that BET inhibition by apabetalone (RVX-208) has effects on multiple biological pathways that underlie disease pathology.

24 Sep 2015

Omeros reports additional positive data from OMS721 Phase 2 trial for treatment of thrombotic microangiopathies

Omeros Corporation today announced additional positive data in the company's Phase 2 clinical trial of OMS721 for the treatment of thrombotic microangiopathies (TMAs). TMAs are a family of rare, debilitating and life-threatening disorders characterized by excessive thrombi (clots) – aggregations of platelets – in the microcirculation of the body's organs, most commonly the kidney and brain.

18 Aug 2015

Omeros' OMS721 granted FDA Fast Track designation for treatment of atypical hemolytic uremic syndrome

Omeros Corporation today announced that the U.S. Food and Drug Administration granted Fast Track designation to OMS721 for the treatment of patients with atypical hemolytic uremic syndrome (aHUS). OMS721 is the company's lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the key regulator of the lectin pathway of the immune system.

23 Jul 2015

Alexion announces that FDA grants ODD to Soliris for Myasthenia Gravis treatment

Alexion Pharmaceuticals (Nasdaq:ALXN) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Soliris® (eculizumab) for the treatment of patients with Myasthenia Gravis (MG), a rare, debilitating neurologic disorder caused by uncontrolled complement activation.

17 Jun 2014

Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today new pre-clinical results with its Development Candidate (DC) for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases.

10 Jun 2014

FDA approves Alexion’s Soliris drug for treatment of atypical hemolytic uremic syndrome

Alexion Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved the Company's supplemental Biologics License Application (sBLA) providing regular approval for Soliris® (eculizumab) for the treatment of adult and pediatric patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).

6 May 2014

Alexion Pharmaceuticals obtains orphan drug designation from EC for Soliris

Alexion Pharmaceuticals, Inc. today announced that the European Commission has granted an orphan drug designation (ODD) to Soliris (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of graft rejection following solid organ transplantation. Graft rejection can cause severe injury to the transplanted organ and is a significant barrier to successful transplantation.

23 Apr 2014

FDA clears Omeros' OMS721 IND for inhibition of complement‑mediated TMAs

Omeros Corporation today announced that its Investigational New Drug Application (IND) to evaluate OMS721 for the inhibition of complement‑mediated thrombotic microangiopathies (TMAs) has been cleared by the U.S. Food and Drug Administration.

3 Apr 2014

Machaon Diagnostics' aHUS Genetic Panel confirms atypical Hemolytic Uremic Syndrome in 48 hours

Machaon Diagnostics today announced the availability of its "aHUS Genetic Panel" (patent pending) to genetically confirm atypical Hemolytic Uremic Syndrome (aHUS) with a turnaround time of 48 hours. The 48 hour timeframe is a dramatic advancement over other approaches which offer results in 4-13 weeks.

1 Apr 2014

Omeros submits investigational new drug to initiate OMS721 Phase 2 clinical program

Omeros Corporation today announced that it has submitted to the U.S. Food and Drug Administration (FDA) an investigational new drug (IND) application to initiate the Phase 2 clinical program for OMS721, the company's lead human monoclonal antibody targeting mannan-binding lectin‑associated serine protease-2 (MASP-2), the key regulator of the lectin pathway of the immune system.

4 Mar 2014