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Related terms: Amyloidosis, primary, Amyloidosis, Secondary Systemic

First Device Approved for Dialysis-Related Amyloidosis

Posted 6 Mar 2015 by

FRIDAY, March 6, 2015 – The U.S. Food and Drug Administration has approved the first device to treat dialysis-related amyloidosis (DRA), a complication of dialysis used to treat kidney failure. DRA is a rare, yet chronic, condition caused by blood buildup of a protein called beta 2-microglobulin. It occurs most often among people aged 60 or older who have been on dialysis for more than five years, the FDA said in a news release. Symptoms if the protein spreads to the bones may include painful and stiff joints, bone cysts and torn tendons and ligaments. The newly approved device, the Lixelle Beta 2-microglobulin Apheresis Column, removes the protein from the blood using porous cellulose beads. It was evaluated among 100 clinical trial patients in Japan. The most common side effects noted were temporary low blood pressure and a drop in red blood cell count. The FDA ordered Japanese ... Read more

Related support groups: Hemodialysis, Peritoneal dialysis, Amyloidosis

Experimental Drug Shows Promise for Rare Genetic Disorder

Posted 29 Aug 2013 by

THURSDAY, Aug. 29 – A new medication appears to be highly effective in combating a heredity-based form of the organ-damaging genetic disorder known as amyloidosis, according to researchers. Amyloidosis refers to a family of more than a dozen diseases in which different types of abnormal proteins called amyloids lodge in major organs and nerves. These amyloids build up to the point that they cause damage and, ultimately, organ failure. The new therapy caused a marked decline in blood-borne levels of an amyloid protein called transthyretin, or TTR, in 32 patients suffering from amyloidosis during one of the two Phase I drug trials, said the study's co-author, Dr. Akshay Vaishnaw, chief medical officer for Alnylam Pharmaceuticals, which developed the medication and funded the trials. "We can dramatically reduce the levels of TTR protein," Vaishnaw said. "In fact, we can reduce them by 94 ... Read more

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Tumor Necrosis Factor Receptor Associated Periodic Syndrome, Periodic Fever Syndrome, Amyloidogenic Transthyretin Amyloidosis, Connective Tissue Disorders

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