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Last updated on April 16, 2014 at 11:02 EDT

Latest Gaucher disease Stories

2014-01-20 10:36:30

What causes brain damage and inflammation in severe cases of Gaucher disease? Little is known about the events that lead to brain pathology in some forms of the disease, and there is currently no treatment available – a bleak outlook for sufferers and their families. Now, scientists at the Weizmann Institute of Science have discovered a new cellular pathway implicated in Gaucher disease. Their findings, published today in Nature Medicine, may offer a new therapeutic target for the...

2012-06-28 02:27:35

NYON, Switzerland, June 28, 2012 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today presented new data that show VPRIV(R) (velaglucerase alfa for injection), the company's enzyme replacement therapy for type 1 Gaucher disease, significantly improved selected markers of Gaucher-related bone disease in patients. These data were presented at the European Working Group on Gaucher Disease (EWGGD) meeting held in Paris, France,...

2011-12-19 08:00:00

EAST SETAUKET, N.Y., Dec. 19, 2011 /PRNewswire/ -- An article in the early edition (December 12, 2011) of the Proceedings of the National Academy of Sciences USA, reported that Lixte's investigational drug, LB-205, was shown to have therapeutic potential in a laboratory model of Gaucher disease (http://www.pnas.org/content/early/2011/12/08/1119181109.full.pdf+html). John Kovach, MD, founder and president of Lixte said, "This is a novel use of the class of drugs known as histone...

2011-12-06 06:30:00

CARMIEL, Israel, Dec. 6, 2011 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE-AMEX: PLX, TASE: PLX), announced today that it received notification from the U.S. Food and Drug Administration (FDA) that the FDA has extended the Prescription Drug User Fee Act (PDUFA) goal date of the New Drug Application (NDA) for taliglucerase alfa to May 1, 2012, a three-month extension from the previous PDUFA date of February 1, 2012. Taliglucerase alfa is the Company's proprietary...

2011-12-01 01:00:00

CARMIEL, Israel, Dec. 1, 2011 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE-AMEX:PLX, TASE:PLX), announced today that the Irish Medicines Board (IMB) has completed a successful GMP (Good Manufacturing Practice) audit of the Company's manufacturing facility in Carmiel, Israel, and has issued a Certificate of GMP Compliance of a Manufacturer for the facility. The IMB Certificate is accepted by all health authorities in the European Union (EU) under the EU's centralized marketing...

2011-09-08 07:00:00

CARMIEL, Israel, Sept. 8, 2011 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE-AMEX: PLX, TASE: PLX), today announced that an article entitled "Pivotal Trial with Plant-Cell-Expressed Recombinant Glucocerebrosidase, taliglucerase alfa, a Novel Enzyme Replacement Therapy for Gaucher Disease" has been published in Blood, the Journal of the American Society of Hematology. The full article can be found on the American Society of Hematology's website at...

2011-08-17 06:47:00

CARMIEL, Israel, Aug. 17, 2011 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE-AMEX:PLX, TASE:PLX), announced today that the U.S. Food & Drug Administration (FDA) has accepted for review the resubmission of the taliglucerase alfa New Drug Application (NDA) following the Company's receipt of a Complete Response Letter (CRL) in February 2011. The FDA deemed the resubmission a class 2, or 6-month, response and established February 1, 2012 as the Prescription Drug User Fee Act (PDUFA)...

2011-08-01 07:00:00

CARMIEL, Israel, Aug. 1, 2011 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE-AMEX:PLX, TASE:PLX), announced today that it has submitted its reply to the Complete Response Letter issued in February 2011 by the U.S. Food and Drug Administration (FDA) after its review of the Company's New Drug Application (NDA) for taliglucerase alfa. Taliglucerase alfa, the Company's proprietary plant-cell expressed form of glucocerebrosidase (GCD), is in development for the treatment of Gaucher...

2011-06-23 21:52:38

Pathway disrupted in Gaucher disease affects Parkinson's-associated protein deposits, suggests novel therapeutic strategy Massachusetts General Hospital investigators appear to have found the mechanism behind a previously reported link between the rare genetic condition Gaucher disease and the common neurodegenerative disorder Parkinson's disease. In a report to appear in the July 8 issue of Cell and receiving early online release, they describe how disruption of the molecular pathway that...

2011-06-15 06:30:00

CARMIEL, Israel, June 15, 2011 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE-AMEX: PLX, TASE: PLX), announced today that Brazil's National Health Surveillance Agency ("Agencia Nacional de Vigilancia Sanitaria" or "ANVISA") has completed a successful GMP (Good Manufacturing Practice) audit of the Company's manufacturing facility in Carmiel, Israel, and determined that the facility is acceptable. The audit was performed as part of the Brazilian Ministry of Health's evaluation of the...