Latest Familial amyloid neuropathy Stories

2014-05-13 08:31:03

- Greater than 100-fold Allele-specific silencing of V30M variant for the treatment of Familial Amyloid Polyneuropathy (TTR-FAP) - SAN DIEGO, May 13, 2014 /PRNewswire/ -- Arcturus Therapeutics, Inc., a leading RNA medicines company pursuing rare diseases, today announced the nomination of its second pipeline program targeting the TTR V30M variant using UNA(TM)-mediated RNAi allele-specific silencing for the treatment of TTR-mediated familial amyloid polyneuropathy (FAP). Additional...

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,...

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...

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...

Word of the Day
  • The navel or umbilicus.
  • In Greek archaeology: A central boss, as on a shield, a bowl, etc.
  • A sacred stone in the temple of Apollo at Delphi, believed by the Greeks to mark the 'navel' or exact center-point of the earth.
'Omphalos' comes from the ancient Greek.