Latest Transthyretin Stories

CARLSBAD, Calif., Feb. 19, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $7.5 million milestone payment from GlaxoSmithKline (GSK) related to the initiation of a Phase 2/3 clinical study for ISIS-TTR(Rx). ISIS-TTR(Rx )is an antisense drug in development with GlaxoSmithKline (GSK) for the treatment of transthyretin (TTR) amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve...

CARLSBAD, Calif., Dec. 13, 2012 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that the United States Food and Drug administration has granted ISIS-TTR(Rx) fast track designation for the treatment of familial amyloid polyneuropathy (FAP). ISIS-TTR(Rx )is an antisense drug in development with GlaxoSmithKline (GSK) for the treatment of transthyretin (TTR) amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral...

CARLSBAD, Calif., Nov. 5, 2012 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that Isis and GlaxoSmithKline (NYSE: GSK) have agreed to amend the clinical development plan and financial terms relating to ISIS-TTR(Rx) to reflect the accelerated development plan for the drug. ISIS-TTR(Rx) is an antisense drug in development with GSK for the treatment of TTR amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral...

Neutrophil extracellular traps and amyloidosis aggravation Amyloidosis is a group of clinical syndromes characterized by deposits of amyloid fibrils throughout the body. These fibrils are formed by aggregates of proteins that have not been properly folded. Deposits of amyloid fibrils are found in a number of diseases, including Alzheimer's and Parkinson's diseases and type-2 diabetes. The amyloid deposits can be localized, as in the brain of Alzheimer's patients, or found spread through...

Scientists from The Scripps Research Institute and Pfizer Inc. have published a new study showing how a new drug called tafamidis (Vyndaqel®) works. Tafamidis, approved for use in Europe and currently under review by the US Food and Drug Administration (FDA), is the first medication approved by a major regulatory agency to treat an amyloid disease, a class of conditions that include Alzheimer's. Tafamidis treats a deadly nerve disease caused by transthyretin (TTR) amyloid fibril...

CARLSBAD, Calif., Jan. 5, 2012 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today results from a Phase 1 study with ISIS-TTRRx. The results demonstrated that treatment with ISIS-TTRRx produced dose-dependent statistically significant reductions of greater than 80 percent in transthyretin (TTR) protein. In this study, ISIS-TTRRx was generally well tolerated with no significant adverse events. ISIS-TTRRx is an antisense drug in development with...

CARLSBAD, Calif., May 19, 2011 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $5 million milestone payment from GlaxoSmithKline (GSK) related to the initiation of a Phase 1 clinical study for ISIS-TTRRx. ISIS-TTRRx is designed to treat transthyretin amyloidosis, a severe and rare genetic disease characterized by slow degeneration of peripheral nerve and/or heart tissues. ISIS-TTRRx is the first antisense drug to enter development under Isis'...

As researchers try to understand how a particular protein sets the stage for the toxic plaques that wreak havoc on the brains of people with Alzheimer's disease, they face a dilemma.This protein, called the amyloid precursor protein, not only begins the damaging process that results in Alzheimer's, it also has a critical biological function in brain development and in the synapse (the junction between nerve cells or nerve and muscle cells). To figure out how this protein causes Alzheimer's,...

NEW YORK and CAMBRIDGE, Mass., Sept. 1 /PRNewswire-FirstCall/ -- Pfizer Inc. (NYSE: PFE) and FoldRx Pharmaceuticals, Inc., a privately held drug discovery and clinical development company, today announced that they have entered into an agreement under which Pfizer will acquire FoldRx. (Logo: http://photos.prnewswire.com/prnh/20100416/PFIZERLOGO ) (Logo: http://www.newscom.com/cgi-bin/prnh/20100416/PFIZERLOGO ) (Logo: http://photos.prnewswire.com/prnh/20100901/NY58014LOGO ) (Logo:...

 Discoveries by Scripps Research Institute scientists have led to a promising new drug candidate"”the first in its class"”for patients with a genetic protein-misfolding disease. In results announced by the biopharmaceutical firm FoldRx Pharmaceuticals, Inc. today, the new drug tafamidis significantly halts disease progression for patients with a disease called Transthyretin (TTR) amyloid polyneuropathy (ATTR-PN)."I'm very excited by the prospect of a drug to help patients...