Gaucher's Disease News and Research

RSS

Gaucher disease is an inherited metabolic disorder in which harmful quantities of a fatty substance called glucocerebroside accumulate in the spleen, liver, lungs, bone marrow, and sometimes in the brain.

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

Mutations in the glucocerebrosidase gene are risk factors for Parkinson's disease

People with a certain gene mutation are more likely to get Parkinson's disease before the age of 50 compared to those without the gene abnormality, according to a study published in the September 18, 2007, issue of Neurology, the medical journal of the American Academy of Neurology.

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

Findings could lead to a new approach in diagnosing and treating Alzheimer’s disease

The human body has its own defense against brain aging: the innate immune system, which helps to clean the brain of amyloid-beta waste products. However, UCLA researchers discovered that some patients with Alzheimer’s disease have an immune defect making it difficult to clean away these wastes.

From MP Biomedicals, LLC 10 Jun 2005

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