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EMA and FDA propose new approach for Gaucher disease drug development
The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have   proposed   new approaches to the development of medicine to treat   Gaucher disease. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - July 4, 2017 Category: Pharmaceuticals Source Type: news

23andMe is back: FDA allows marketing of genetic health risk tests
[Image courtesy of 23andMe]FDA this month allowed genetic testing company 23andMe to market genetic health risk tests for 10 diseases and conditions including Parkinson’s disease and late-onset Alzheimer’s disease. The de novo premarket review authorization, announced April 6, also included celiac disease, which results in the inability to digest gluten; alpha-1 antitrypsin deficiency, which raises the risk of lung and liver disease; early-onset primary dystonia, a movement disorder; factor XI deficiency, a blood clotting disorder; Gaucher disease type 1, a disorder involving organs and tissue; glucose-6-p...
Source: Mass Device - April 21, 2017 Category: Medical Equipment Authors: Chris Newmarker Tags: Business/Financial News Diagnostics Food & Drug Administration (FDA) Genomics Molecular Diagnostics News Well Regulatory/Compliance 23andme 23andMe Inc. MedTech Source Type: news

Avrobio launches gene therapy program for Gaucher disease
Cambridge, Mass.-based Avrobio said today that it expanded its pipeline to include a preclinical program focused on a gene therapy for Gaucher disease. The investigational gene therapy makes use of a patient’s blood stem cells, genetically modifying them with a fully functional copy of the faulty gene responsible for the glucosylceramide accumulation that is characteristic of Gaucher disease. Avrobio licensed the technology from Lund University’s Dr. Stefan Karlsson, but did not disclose the terms of the agreement. Get the full story at our sister site, Drug Delivery Business News. The post Avrob...
Source: Mass Device - March 22, 2017 Category: Medical Equipment Authors: Sarah Faulkner Tags: Research & Development Stem Cells Wall Street Beat Avrobio Source Type: news

Study suggests new therapy for Gaucher disease
Scientists propose that blocking a molecule that drives inflammation and organ damage in Gaucher, and maybe other lysosomal storage diseases, as a possible treatment with fewer risks and lower costs than current therapies. The team conducted the study in mouse models of lysosomal storage disease and in cells from blood samples donated by people with Gaucher disease. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - February 22, 2017 Category: Science Source Type: news

Nature study suggests new therapy for Gaucher disease
(Cincinnati Children's Hospital Medical Center) Scientists propose in Nature blocking a molecule that drives inflammation and organ damage in Gaucher, and maybe other lysosomal storage diseases, as a possible treatment with fewer risks and lower costs than current therapies. Reporting their data Feb. 22, the international research team conducted the study in mouse models of lysosomal storage disease and in cells from blood samples donated by people with Gaucher disease. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - February 22, 2017 Category: Cancer & Oncology Source Type: news

New Topic Page: Lipid Metabolism Disorders
Visit the new MedlinePlus Health Topic page on Lipid Metabolism Disorders. Lipid metabolism disorders, such as Gaucher disease and Tay-Sachs disease, involve lipids. Lipids are fats or fat-like substances... (Source: What's New on MedlinePlus)
Source: What's New on MedlinePlus - August 24, 2016 Category: Consumer Health News Source Type: news

Molecule may impact Gaucher, Parkinson ’s disease
Scientists identified a molecule that restores the activity of a Gaucher disease-related enzyme and decreases alpha-synuclein protein buildup —a hallmark of Parkinson’s disease. (Source: NIH Research Matters from the National Institutes of Health (NIH))
Source: NIH Research Matters from the National Institutes of Health (NIH) - July 26, 2016 Category: Consumer Health News Source Type: news

National Gaucher Foundation Commends NIH for Continued Research on...
National Gaucher Foundation today commended National Institutes of Health scientist Dr. Ellen Sidransky for her tireless work on Gaucher disease. Her research, which was published on July 12,...(PRWeb July 18, 2016)Read the full story at http://www.prweb.com/releases/2016/07/prweb13558012.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - July 19, 2016 Category: Pharmaceuticals Source Type: news

Researchers advance treatment possibilities for Gaucher, Parkinson's
Bethesda, Md., Mon., July 12, 2016 - NIH researchers have identified and tested a molecule that shows promise as a possible treatment for the rare Gaucher disease and the more common Parkinson's disease. These findings demonstrate how insights from a rare disorder can have direct relevance to the treatment of more common disorders. The findings were published July 12, 2016 in The Journal of Neuroscience. (Source: NHGRI Press Releases)
Source: NHGRI Press Releases - July 12, 2016 Category: Genetics & Stem Cells Source Type: news

Researchers advance treatment possibilities for Gaucher, Parkinson's
National Institutes of Health researchers have identified and tested a molecule that shows promise as a possible treatment for the rare Gaucher disease and the more common Parkinson's disease. These findings demonstrate how insights from a rare disorder can have direct relevance to the treatment of more common disorders. Gaucher disease affects an estimated 1 in 50,000 to 1 in 100,000 people in the general population. Parkinson's disease affects more than 1 million people in North America and 7-10 million people worldwide. The findings were published July 12 in The Journal of Neuroscience. (Source: NHGRI Homepage Highlights)
Source: NHGRI Homepage Highlights - July 12, 2016 Category: Genetics & Stem Cells Source Type: news

The National Gaucher Foundation Mourns the Loss of Dr. Roscoe Brady
The National Gaucher Foundation today mourns the passing of Dr. Roscoe Owen Brady, M.D., a pioneer in medicine and the father of enzyme therapy for lysosomal diseases, including Gaucher disease.(PRWeb June 15, 2016)Read the full story at http://www.prweb.com/releases/2016/06/prweb13490867.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - June 15, 2016 Category: Pharmaceuticals Source Type: news

What Doctors Got Wrong About 'Good' Cholesterol
A new genetic study published in the journal Science suggests that contrary to the conventional wisdom, high levels of good cholesterol aren't necessarily heart-protective for everyone. "Twenty years ago, if you had high bad cholesterol and high good cholesterol, doctors said don’t worry about it -- one offsets the other," Dr. Scott Wright, a cardiologist at the Mayo Clinic, who wasn't involved in the study, told The Huffington Post."I never really bought that, and time has proven my skepticism to be correct. You can have a heart attack despite having a high level of good cholesterol." D...
Source: Science - The Huffington Post - March 17, 2016 Category: Science Source Type: news

Lysolipid antigens prominent in MGUS and myeloma
Clonal immunoglobulin reactive to lyso-glucosylceramide (LGL1), which is elevated in Gaucher disease, was found in patients with Gaucher disease as well as in one third of patients with sporadic... (Source: Family Practice News)
Source: Family Practice News - February 10, 2016 Category: Primary Care Source Type: news

Gaucher disease may protect against Parkinson's disease-related color visual impairment
Parkinson's Disease (PD) patients have a five-fold greater risk of carrying genetic mutations in the β-glucocerebrosidase gene (GBA), which are commonly associated with Gaucher disease (GD). (Source: Parkinson's Disease News From Medical News Today)
Source: Parkinson's Disease News From Medical News Today - November 11, 2015 Category: Neurology Tags: Parkinson's Disease Source Type: news

Gaucher disease may protect against Parkinson's disease-related color visual impairment
Parkinson’s Disease (PD) patients have a five-fold greater risk of carrying genetic mutations in the ?-glucocerebrosidase gene (GBA), which are commonly associated with Gaucher disease (GD). Patients with both PD and GD tend to experience earlier onset of PD and more serious cognitive changes than PD patients without the mutations. A new study suggests that GD or the presence of GBA mutations may actually shield patients from deficiency in visual color discrimination, which is a hallmark of PD. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - November 10, 2015 Category: Science Source Type: news

Gaucher disease may protect against Parkinson's disease-related color visual impairment
(IOS Press) Patients with both PD and GD tend to experience earlier onset of PD and more serious cognitive changes than PD patients without the mutations. A new study published in the Journal of Parkinson's Disease suggests that GD or the presence of GBA mutations may actually shield patients from deficiency in visual color discrimination, which is a hallmark of PD. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 10, 2015 Category: Global & Universal Source Type: news

Sanofi says Cerdelga trial shows promise
PARIS (Reuters) - French pharmaceutical group Sanofi said on Tuesday a trial treatment with its Cerdelga drug for patients with type 1 Gaucher disease, a rare genetic disorder, showed promise. (Source: Reuters: Health)
Source: Reuters: Health - February 17, 2015 Category: Consumer Health News Tags: healthNews Source Type: news

Eliglustat (Cerdelga) Approved in Europe for Gaucher DiseaseEliglustat (Cerdelga) Approved in Europe for Gaucher Disease
The oral glucosylceramide analog is indicated for long-term treatment of adults with type 1 Gaucher disease. International Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - January 23, 2015 Category: Consumer Health News Tags: Hematology-Oncology News Alert Source Type: news

Genzyme’s Cerdelga receives European approval to treat Gaucher disease type 1
Sanofi subsidiary Genzyme has received marketing approval from European Commission (EC) for its Cerdelga (eliglustat) capsules to treat adult patients with Gaucher disease type 1. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - January 23, 2015 Category: Pharmaceuticals Source Type: news

Drug used for another disease slows progression of Parkinson's
A drug being evaluated to treat an entirely different disorder helped slow the progression of Parkinson’s disease in mice, a team of researchers has reported. Their study found that the drug, AT2101, which has also been studied for Gaucher disease, improved motor function, stopped inflammation in the brain and reduced levels of alpha-synuclein, a protein critically involved in Parkinson's. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - October 8, 2014 Category: Science Source Type: news

UCLA researchers find that drug used for another disease slows progression of Parkinson’s
A new study from UCLA found that a drug being evaluated to treat an entirely different disorder helped slow the progression of Parkinson’s disease in mice. The study, published in the October edition of the journal Neurotherapeutics, found that the drug, AT2101, which has also been studied for Gaucher disease, improved motor function, stopped inflammation in the brain and reduced levels of alpha-synuclein, a protein critically involved in Parkinson’s. Although the exact cause of Parkinson’s is unknown, evidence points to an accumulation of alpha-synuclein, which has been found to be common to all people w...
Source: UCLA Newsroom: Health Sciences - October 8, 2014 Category: Universities & Medical Training Source Type: news

FDA Clears Taliglucerase Alfa for Kids With Gaucher Disease FDA Clears Taliglucerase Alfa for Kids With Gaucher Disease
Taliglucerase alfa is now indicated for long-term enzyme replacement therapy for children and adults with type 1 Gaucher disease. FDA Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - September 2, 2014 Category: Consumer Health News Tags: Hematology-Oncology News Alert Source Type: news

Pfizer and Protalix’s Elelyso receives FDA approval to treat type 1 Gaucher disease
Pfizer and Protalix BioTherapeutics have secured approval from the US Food and Drug Administration (FDA) for Elelyso (taliglucerase alfa) to treat paediatric patients with type 1 Gaucher disease. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - August 31, 2014 Category: Pharmaceuticals Source Type: news

New Drug Approved for Gaucher Disease Type 1 (FREE)
By the Editors The FDA has approved eliglustat (Cerdelga) to treat … (Source: Physician's First Watch current issue)
Source: Physician's First Watch current issue - August 21, 2014 Category: Primary Care Source Type: news

FDA Clears Eliglustat (Cerdelga) for Gaucher DiseaseFDA Clears Eliglustat (Cerdelga) for Gaucher Disease
The new drug proved as effective as enzyme replacement therapy in treating the rare genetic disease in a clinical trial. FDA Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - August 19, 2014 Category: Consumer Health News Tags: Hematology-Oncology News Alert Source Type: news

FDA approves Sanofi's Gaucher disease drug Cerdelga
(Reuters) - The U.S. Food and Drug Administration said on Tuesday it has approved Sanofi SA's drug Cerdelga to treat patients with type 1 Gaucher disease, a rare genetic disorder. (Source: Reuters: Health)
Source: Reuters: Health - August 19, 2014 Category: Consumer Health News Tags: healthNews Source Type: news

FDA approves new drug to treat a form of Gaucher disease
The U.S. Food and Drug Administration today approved Cerdelga (eliglustat) for the long-term treatment of adult patients with the Type 1 form of Gaucher disease, a rare genetic disorder. (Source: Food and Drug Administration)
Source: Food and Drug Administration - August 19, 2014 Category: American Health Source Type: news

Glucocerebrosidase Is Shaking Up the SynucleinopathiesGlucocerebrosidase Is Shaking Up the Synucleinopathies
Recent progress in unraveling the mechanisms involving the enzyme glucocerebrosidase is not only advancing therapy for Gaucher disease, but may impact understanding of Parkinson's disease as well. Brain (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - July 25, 2014 Category: Consumer Health News Tags: Neurology & Neurosurgery Journal Article Source Type: news

Protalix Announces New Data on ELELYSO(TM) (taliglucerase alfa) Presented at the European Working Group on Gaucher Disease 2014 11th Meeting
CARMIEL, Israel, June 27, 2014 -- (Healthcare Sales & Marketing Network) -- Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (PLX.TA), announced today that new clinical data on ELELYSOTM (taliglucerase alfa) will be presented at the European Working Group on ... BiopharmaceuticalsProtalix BioTherapeutics, ELELYSO, taliglucerase alfa, Gaucher Disease (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - June 27, 2014 Category: Pharmaceuticals Source Type: news

Gaucher Disease Points Way to Possible New Treatment for Parkinson's
Source: Parkinson's Disease Foundation Related MedlinePlus Pages: Gaucher's Disease, Parkinson's Disease (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - June 26, 2014 Category: Consumer Health News Source Type: news

Protalix starts Phase II trial of oral GCD to treat Gaucher disease
Israel-based Protalix BioTherapeutics has started enrolling patients in its Phase IIa clinical trial of oral glucocerebrosidase (GCD), or oral GCD (PRX-112) for treatment of Gaucher disease. (Source: Drug Development Technology)
Source: Drug Development Technology - June 19, 2014 Category: Pharmaceuticals Source Type: news

A Product For Gaucher Disease And A Plant-Cell For Protein Production – Protalix Biotherapeutics Is On The Rise
Protalix has a unique plant-cell platform for therapeutic protein production and an approved product, Elelyso/Uplyso (taliglucerase alfa), for Gaucher disease. In the company’s pipeline is a chemically modified version of the recombinant alpha-Galactosidase-A protein for Fabry disease (Phase 1/2), an oral glucocerebrosidase (GCD) enzyme replacement therapy for Gaucher disease (Phase 1), an oral anti-TNF (tumor necrosis factor) fusion protein for autoimmune/inflammatory conditions (preclinical), and a human deoxyribonuclease I (DNase I) for cystic fibrosis (preclinical), and other enzyme replacement therapies in early...
Source: Pharmaceutical Online News - May 30, 2014 Category: Pharmaceuticals Source Type: news

Protalix gets marketing approval in Australia for ELELYSO to treat Gaucher disease
Israel-based biopharmaceutical firm Protalix BioTherapeutics has received approval from the Australian Therapeutic Goods Administration (TGA) for ELELYSO (taliglucerase alfa) for injection, as a long-term enzyme replacement therapy for treatment of G… (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - May 22, 2014 Category: Pharmaceuticals Source Type: news

People with Gaucher Disease at Seven to Nine Percent Risk of Developing Parkinson's
Source: Parkinson's Disease Foundation Related MedlinePlus Pages: Gaucher's Disease, Parkinson's Disease (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - May 21, 2014 Category: Consumer Health News Source Type: news

New hope for Gaucher patients with brain pathology
Gaucher disease, a genetic disorder prevalent among Ashkenazi Jews, is devastating for sufferers and their families. Now, scientists have discovered a new cellular pathway implicated in the disease. Their findings may offer a new therapeutic target for treatment of Gaucher and related disorders. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - January 27, 2014 Category: Science Source Type: news

New hope for Gaucher patients
(Weizmann Institute of Science) Weizmann Institute scientists have discovered a new cellular pathway implicated in Gaucher disease. Their findings may offer a new therapeutic target for the management of this disease, as well as other related disorders. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - January 19, 2014 Category: Biology Source Type: news

Gaucher disease link to Parkinson's disease may be explained by overworked cellular machines
Scientists have identified the biological pathway that explains Gaucher disease (GD) patients' higher risk for developing Parkinson's disease (PD), according to research presented at the American Society for Cell Biology annual meeting in New Orleans.GD is a hereditary disease in which the genes that encode the enzyme glucocerebrosidase are mutated, resulting in a life-threatening build-up of lipids, fatty substances, in cells. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - December 23, 2013 Category: Consumer Health News Tags: Parkinson's Disease Source Type: news

Sanofi Gaucher pill application gets priority FDA review
PARIS (Reuters) - French drugmaker Sanofi said U.S. authorities had decided to carry out a priority review of its experimental pill for patients with Gaucher disease. (Source: Reuters: Health)
Source: Reuters: Health - December 11, 2013 Category: Consumer Health News Tags: healthNews Source Type: news

What Is Gaucher's Disease? What Causes Gaucher's Disease?
Gaucher's (Gaucher) disease, also known as glucocerebrosidase deficiency, occurs when a certain lipid, glucosylceramide, accumulates in the bone marrow, lungs, spleen, liver and sometimes the brain. Patients with Gaucher's disease have a hereditary deficiency of glucocerebrosidase, an enzyme that helps the body process glucosylceramide. Glucosylceramide is also known as glucocerebroside. When glucocerebrosidase is faulty, the lipid (fat) glucosylceramide builds up, especially in the macrophages. Macrophage is a type of white blood cell... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - July 15, 2013 Category: Consumer Health News Tags: Endocrinology Source Type: news

Effect of Imiglucerase on Metabolism in Gaucher DiseaseEffect of Imiglucerase on Metabolism in Gaucher Disease
Enzyme replacement therapy is the gold standard treatment for Gaucher disease. What impact does ERT have on growth and metabolism of GD type 1 patients? Nutrition and Metabolism (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - July 1, 2013 Category: Consumer Health News Tags: Pathology & Lab Medicine Journal Article Source Type: news

Gaucher Patients Often Develop Parkinson's Disease Due To Defective Cellular Waste Removal
Gaucher disease causes debilitating and sometimes fatal neurodegeneration in early childhood. Recent studies have uncovered a link between the mutations responsible for Gaucher disease and an increased risk of developing Parkinson's disease later in life. New research published online in the Cell Press journal Cell Metabolism indicates that the neurodegeneration found in Gaucher disease stems from defects in processes that break down and remove unwanted material from cells... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - May 27, 2013 Category: Consumer Health News Tags: Neurology / Neuroscience Source Type: news

Elelyso (Taliglucerase Alfa) for Treating Gaucher Disease
Elelyso (taliglucerase alfa) is an intravenous drug indicated for the treatment of type one Gaucher disease. The drug was jointly developed by Protalix BioTherapeutics and Pfizer. (Source: Drug Development Technology)
Source: Drug Development Technology - April 17, 2013 Category: Pharmaceuticals Source Type: news

Horizon scanning: Phase III data for eliglustat for Gaucher disease type 1
Source: BioSpace Area: News BioSpace has reported on new Phase III data for eliglustat tartrate, an investigational oral therapy for Gaucher disease type 1.   The randomised, double-blind, placebo-controlled ENGAGE study evaluated the safety and efficacy of eliglustat tartrate in 40 treatment-naïve patients with Gaucher disease type 1. The primary endpoint was spleen size - spleen volume decreased by a mean of 28% in patients treated with eliglustat tartrate and increased by a mean of 2% in those treated with placebo (p
Source: NeLM - News - February 18, 2013 Category: Drugs & Pharmacology Source Type: news

Sanofi says Gaucher pill studies meet goals
PARIS (Reuters) - Sanofi said its experimental pill for Gaucher disease met its main targets in two late-stage studies, which the French drugmaker will use to bolster its case for regulatory approval. (Source: Reuters: Health)
Source: Reuters: Health - February 15, 2013 Category: Consumer Health News Tags: healthNews Source Type: news

Oral Drug for Gaucher Disease PromisingOral Drug for Gaucher Disease Promising
Sanofi said its experimental oral drug for Gaucher disease, eliglustat tartrate, met its main targets in two late-stage studies, which the French drugmaker will use to bolster its case for regulatory approval. Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - February 15, 2013 Category: Consumer Health News Tags: Family Medicine/Primary Care News Source Type: news

Genzyme Announces Positive New Data from Two Phase 3 Studies for Oral Eliglustat Tartrate for Gaucher Disease
CAMBRIDGE, Mass.--(Healthcare Sales & Marketing Network)--Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced positive new data from the Phase 3 ENGAGE and ENCORE studies of eliglustat tartrate, its investigational oral therapy for Gau... BiopharmaceuticalsGenzyme, Sanofi, eliglustat, Gaucher disease (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - February 15, 2013 Category: Pharmaceuticals Source Type: news

Genzyme Announces Positive New Data From Two Phase 3 Studies For Oral Eliglustat Tartrate For Gaucher Disease
Genzyme, a Sanofi company, recently announced positive new data from the Phase 3 ENGAGE and ENCORE studies of eliglustat tartrate, its investigational oral therapy for Gaucher disease type 1. (Source: Pharmaceutical Online News)
Source: Pharmaceutical Online News - February 15, 2013 Category: Pharmaceuticals Source Type: news

Webinar-Between Thought and Therapy: Translating Neurobiology Research into Treatments
This webinar will explore how translational neurobiology research is being conducted in the Intramural Research Program of the NIH in disorders as diverse as depression, age-related macular degeneration, and Gaucher's disease. Panelists all conduct translational research across the full bench-to-bedside continuum, with the ultimate goal of developing novel paradigms for the treatment of a range of diseases and improving the quality of life for patients. (Source: News from NEI)
Source: News from NEI - January 31, 2013 Category: Opthalmology Tags: News and Events Source Type: news

Inside The Pricing Of A $300,000-A-Year Drug
Last night, NPS Pharmaceuticals announced that it was pricing Gattex, its drug for short bowel syndrome, at $295,000 per patient per year, about triple what analysts on Wall Street expected. It is the fourth drug approved in 2012 to be priced at more than $200,000 per patient per year. The others are: Kalydeco for cystic fibrosis (maker :Vertex Pharmaceuticals); Elelyso for Gaucher's disease (Protalix and Pfizer); and Juxtapid for homozygous familial hypercholesterolemia (Aegerion Pharmaceuticals.) That represents 10% of the drugs approved by the Food and Drug Administration last year and a 44% increase in the number of su...
Source: Forbes.com Healthcare News - January 3, 2013 Category: Pharmaceuticals Authors: Matthew Herper Source Type: news