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Last updated on April 17, 2014 at 1:21 EDT

Latest Familial amyloid neuropathy Stories

2014-02-14 16:24:52

CARLSBAD, Calif., Feb. 14, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending ISIS-TTR(Rx) for designation as an orphan medicinal product for the treatment of ATTR-Amyloidosis to the European Commission (EC). The opinion will be subject to review by the EC, which ultimately grants the decision on orphan drug designation. ATTR-Amyloidosis, or TTR amyloidosis,...

2013-02-19 08:31:43

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 and/or heart...

2012-12-13 08:26:37

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 nerve...

2012-11-05 08:32:00

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 nerves...

2012-05-30 10:03:52

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...

2012-01-05 07:00:00

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...

2011-05-19 06:00:00

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'...

2010-09-01 07:30:00

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:...

2009-07-22 09:11:00

 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...

2008-11-11 09:00:08

A recent study of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) has enabled a new understanding of the rare and fatal disease. The study explored the diagnosis, progression and mortality of the disease, and resulted in new insight into measures of disease progression as well as potential predictors of survival. Currently there is no available disease modifying pharmacologic therapy for ATTR-CM, and these significant strides in expanding the limited understanding of ATTR-CM will guide...