Gaucher's Disease News and Research

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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.

European scientists set up new therapeutic approaches to tackle gene defects

On 15th April is the 1st International Pompe Disease Day, a campaign to raise awareness of this rare but severe gene defect. Pompe Disease is only one of more than 40 metabolic disorders that mainly affect children under the age of 10, often with devastating consequences.

15 Apr 2014

Research findings may offer new therapeutic target for management of Gaucher diseas

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.

19 Jan 2014

Genzyme granted Priority Review for Cerdelga investigational oral therapy for Gaucher disease

Genzyme, a Sanofi company, announced today that the Food and Drug Administration (FDA) has granted a six-month Priority Review designation to its New Drug Application (NDA) for Cerdelga™ (eliglustat), an investigational oral therapy for adult patients with Gaucher disease type 1.

11 Dec 2013

Renaissance in drug development for rare diseases

Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood drama Lorenzo's Oil, rare diseases are getting unprecedented attention today among drug manufacturers, who are ramping up research efforts and marketing new medicines that promise fuller lives for children and other patients with these heartbreaking conditions.

14 May 2013

Genzyme marks 2013 International Rare Disease Day

Genzyme, a Sanofi company, today announced its support of International Rare Disease Day with a series of initiatives meant to call attention to rare diseases as an important public health issue and to improve rare disease education, research, and treatment.

28 Feb 2013

Positive new data from Genzyme’s eliglustat tartrate Phase 3 studies on Gaucher disease type 1

Genzyme, a Sanofi company, today announced positive new data from the Phase 3 ENGAGE and ENCORE studies of eliglustat tartrate, its investigational oral therapy for Gaucher disease type 1.

15 Feb 2013

Top-line results from Neuraltus’ NP001 Phase 2 study on amyotrophic lateral sclerosis

Neuraltus Pharmaceuticals, a private biopharmaceutical company developing novel therapies for rare neurological disorders, announced today top-line results from the Company's Phase 2 clinical study of NP001 in patients with Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease).

30 Oct 2012

Maryland scientists create stem cell model for Gaucher disease

A new method of using adult stem cells as a model for the hereditary condition Gaucher disease could help accelerate the discovery of new, more effective therapies for this and other conditions such as Parkinson's, according to new research from the University of Maryland School of Medicine.

16 Oct 2012

FDA approves Protalix, Pfizer’s ELELYSO for treatment of type 1 Gaucher disease

Pfizer Inc. and Protalix BioTherapeutics, Inc. announced today that the United States (U.S.) Food and Drug Administration (FDA) approved ELELYSO (taliglucerase alfa) for injection, an enzyme replacement therapy (ERT) for the long-term treatment of adults with a confirmed diagnosis of type 1 Gaucher disease.

2 May 2012

Pfizer receives FDA approval for Elelyso to treat Type 1 Gaucher disease

The U.S. Food and Drug Administration today approved Elelyso (taliglucerase alfa) for long-term enzyme replacement therapy to treat a form of Gaucher disease, a rare genetic disorder.

2 May 2012

New method uses E. coli bacteria to engineer human therapeutic glycoproteins

Escherichia coli - a bacteria considered the food safety bane of restaurateurs, grocers and consumers - is a friend. Cornell University biomolecular engineers have learned to use E. coli to produce sugar-modified proteins for making pharmaceuticals cheaper and faster.

27 Mar 2012

GSK, Angiochem to develop and commercialize LSD treatments

Angiochem announced today a global collaboration with GlaxoSmithKline (GSK) to discover, develop and commercialize treatments for lysosomal storage diseases.

27 Feb 2012

Genzyme announces four-year data from eliglustat tartrate phase 2 trial on Gaucher disease type 1

Genzyme, a Sanofi company, announced today four-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate.

8 Feb 2012

Lixte's LB-205 shows therapeutic potential against Gaucher disease

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.

19 Dec 2011

PDUFA goal date of NDA for Protalix's taliglucerase alfa extended

Protalix BioTherapeutics, Inc., 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.

6 Dec 2011

Four companies receive NIAID contracts to develop broad-spectrum therapeutics

Four companies are to develop broad-spectrum therapeutics-antibiotics, antivirals and an antitoxin-to prevent or treat diseases caused by multiple types of bacteria or viruses, under contracts awarded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.

14 Oct 2011

FDA accepts Protalix taliglucerase alfa NDA resubmission for review

Protalix BioTherapeutics, Inc., 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.

17 Aug 2011

Blood stored in banks may undergo changes and damage

New research provides evidence for significant differences between new and old red blood cells used for transfusions and could provide a cheap, rapid and effective way to monitor the quality of blood supplies.

21 May 2011

New insight into Parkinson's disease

As Parkinson's Awareness Month gets underway, a Canadian-led international study is providing important new insight into Parkinson's disease and paving the way for new avenues for clinical trials.

5 Apr 2011

Shire's Replagal and VPRIV data on Gaucher and Fabry patients presented at ACMG meeting

Shire plc, the global specialty biopharmaceutical company, presented new data for Replagal in patients with Fabry disease, and VPRIV in patients with type 1 Gaucher disease, at the 2011 American College of Medical Genetics' Annual Clinical Genetics Meeting, held in Vancouver, BC, from March 16-20, 2011.

22 Mar 2011