Gaucher Disease News and Research

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Blood vessels around the brain now converted into production/delivery systems

The blood brain barrier is generally considered an obstacle to delivering therapies from the bloodstream to the brain. However, University of Iowa researchers have discovered a way to turn the blood vessels surrounding brain cells into a production and delivery system for getting therapeutic molecules directly into brain cells.

22 Sep 2009

Protalix Biotherapeutics reports completion of its Phase III trial of prGCD

Protalix Biotherapeutics, Inc. (NYSE-Amex: PLX), announced the completion of its pivotal Phase III trial for prGCD, the Company’s proprietary plant-cell expressed recombinant form of glucocerebrosidase (GCD) for the treatment of Gaucher disease. The trial enrolled a total of 31 patients across Europe, North America, South America, Israel and South Africa. No serious adverse events were reported in this trial.

14 Sep 2009

Protalix receives orphan drug designation for prGCD

Protalix BioTherapeutics, Inc. (NYSE-Amex:PLX), announced today that it has received notice from the U.S. Food and Drug Administration (FDA) that the FDA’s Office of Orphan Products Development has granted orphan drug designation to prGCD, the Company’s proprietary plant cell expressed recombinant form of glucocerebrosidase (GCD) for the treatment of Gaucher disease.

8 Sep 2009

Genzyme updates on Cerezyme supply plus 2009 financials

Genzyme Corporation (NASDAQ: GENZ) today provided an update on the impact of Cerezyme® (imiglucerase for injection) supply constraints on patient treatment regimens and on the company’s 2009 financial results. The company’s Fabrazyme® (agalsidase beta) supply constraint period, Fabrazyme dose conservation guidelines for patients, and Fabrazyme financial guidance are unchanged.

11 Aug 2009

One injection of gene therapy spreads through brain in animal study

By targeting a site in a mouse brain well connected to other areas, researchers successfully delivered a beneficial gene to the entire brain - after one injection of gene therapy.

9 Oct 2007

Genzyme receives Japanese marketing approval for Elaprase

Genzyme Corp. has announced that it has received approval to market Elaprase (idursulfase) in Japan for the treatment of Hunter syndrome.

4 Oct 2007

Researchers question genetic screening programs

The study by a team at Shaare Zedek Medical Centre in Jerusalem, is the first to examine what couples actually did when they learned their unborn child had a condition called Gaucher disease, an inherited condition which can be effectively treated.

20 Sep 2007

Study examines implications of genetic screening for disease that can be less serious, treatable

Some couples in Israel whose fetus screened positive for Gaucher disease, which can range from being mild and treatable to being a severe disease, decided to have the pregnancy terminated, raising questions concerning the appropriateness of certain types of genetic screenings, according to a study in the September 19 issue of JAMA.

18 Sep 2007

Novel preclinical gene therapy approach for treating Niemann-Pick disease

Genzyme Corp. has announced the publication of a new study in The Proceedings of the National Academy of Sciences describing a novel preclinical gene therapy approach for treating Niemann-Pick disease.

24 May 2007

Protalix BioTherapeutics receives FDA approval to initiate a phase III trial of prGCD

Protalix BioTherapeutics, Inc. has announced that it has received written notice from the United States Food and Drug Administration (FDA) that it may initiate a Phase III clinical trial in the United States of its lead product candidate, prGCD, a proprietary plant cell expressed recombinant form of human Glucocerebrosidase (GCD), for the treatment of Gaucher Disease, a lysosomal storage disorder in humans.

19 Apr 2007

FDA needs authority to approve generic biologics

AARP called on Congress today to give the Food and Drug Administration (FDA) the authority to approve safe, cost-effective generic versions of often expensive biologic drug therapies.

8 Mar 2007

New genetic clues to dementia with Lewy bodies

Researchers have found that genetic alterations originally identified in people suffering from a rare disease may also be an important risk factor for the second most common form of dementia among the elderly.

17 Jul 2006

Genzyme files Glucocerebrosidase patent infringement

Genzyme announced today that it has filed a lawsuit in the District Court of Tel Aviv alleging that Transkaryotic Therapies Inc. has infringed Genzyme's Israeli patent No. 100,715, which covers certain cell culture processes involved in the manufacture of glucocerebrosidase.

25 Jan 2005

Clinical trial of enzyme replacement therapy for the treatment of Gaucher disease

Transkaryotic Therapies, Inc. today announced that it has initiated a clinical trial to evaluate the safety and clinical activity of GA-GCB, its enzyme replacement therapy for the treatment of Gaucher disease.

26 Apr 2004