Navigation Links
Phase II study of an oral therapy for Gaucher disease yields positive results
Date:5/3/2010

(WASHINGTON, May 3, 2010) Gaucher disease, a rare enzyme deficiency disorder, is one of many conditions with few approved treatment options for patients. In a study published online today in Blood, the journal of the American Society of Hematology, researchers present positive results of a Phase II clinical trial of eliglustat tartrate, an oral therapy in development to treat Gaucher disease.

Gaucher disease is a genetic disorder affecting an estimated 1 in 50,000 to 1 in 100,000 people in the U.S. population. Gaucher disease occurs when a mutation of the glucocerebrosidase gene causes low activity of that enzyme in the body. As a result, harmful fatty substances accumulate in the liver, spleen, bones, and bone marrow, preventing cells and organs from working properly. The primary treatment option is enzyme replacement therapy, which is given intravenously, to break down the accumulated fatty substances.

Eliglustat tartrate is an oral drug currently in development for Gaucher disease type 1 (GD1). As a substrate reduction therapy, the drug decreases the body's production of the fatty substances so they do not accumulate in cells. Based on Phase I trials in healthy volunteers that demonstrated positive initial safety results, the research team initiated a multinational, open-label, single-arm Phase II study of 26 GD1 patients to evaluate the efficacy, safety, and pharmacokinetics (how the body absorbs and processes the drug) of eliglustat tartrate administered orally at 50mg or 100mg doses twice daily. Eligible patients had confirmed Gaucher disease, characterized as enzyme deficiency and a spleen volume at least 10 times greater than normal, plus abnormal values of either platelet count or hemoglobin levels.

"As with many other rare conditions, we strive to develop new options with improved efficacy and safety for our patients, but we also look for options that may be easier on the provider and the patient.In this case, we we
'/>"/>

Contact: Wendy Stokes
webmaster@hematology.org
202-776-0544
American Society of Hematology
Source:Eurekalert

Page: 1 2

Related medicine news :

1. New phase II study supports potential of gs-9450 as new treatment option for steatohepatitis
2. Pilot Testing Phase Ends; Expansion Of Sales Force Begins
3. Southampton scientists begin Phase II patient trial for new asthma treatment
4. Intellicate Announces New “Phased Return to Work” (PRtW) Service
5. Askthedoctor.com Makes Medical Information More Accessible to the Public by Completing Phase 1 of US Expansion
6. Nuvo Research enrolls first patient in Phase 2 WF10 allergic rhinitis trial
7. Poniard Pharmaceuticals Presents Positive Survival Data from a Phase 2 Clinical Study of Picoplatin in Metastatic Prostate Cancer at the 2010 American Society of Clinical Oncology Genitourinary Cancers Symposium
8. New High Purity Immunoglobulin Enters Phase III in Europe and the US
9. Medical Imaging Northwest Completes Phase I of Its Healthcare IT Integration to Maintain Patient Care Improvements
10. NASA selects DNA Medicine Institutes Nanoscale Diagnostics Technology for Phase II SBIR
11. PRA International Rolls out Client Access to Oracle(R) RDC for Early Phase Studies
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/22/2014)... there are only 24 hours in a day, that doesn’t seem ... time within their busy schedules for exercise and fitness. Fortunately, one ... get in shape. Here are five ways you can reach your ... Up Your Commute , Consider riding a bike or taking the ... daily routine. If you must use your car to get to ...
(Date:10/22/2014)... WALTHAM, MA (PRWEB) October 22, 2014 ... developing new solutions for the treatment of bacterial infections, ... J. Sinskey has joined its board of directors. ... Microbiology and Engineering Systems at The Massachusetts Institute of ... the MIT faculty since 1968. Dr. Sinskey also holds ...
(Date:10/22/2014)... News) -- Two sisters in high school have developed ... and Medha Krishen use electronic stethoscopes, which electronically amplify ... breathing patterns or heartbeats. Ilina, a senior at ... find a way to detect early lung damage in ... stethoscope, Ilina recorded one breath cycle each from 16 ...
(Date:10/22/2014)... TUESDAY, Oct. 21, 2014 (HealthDay News) -- Measures taken ... plantation in Ebola-ravaged Liberia may have limited the spread ... researchers report. The Firestone Natural Rubber Co. provides ... and residents of nearby densely populated communities. Between ... and suspected Ebola cases among those 80,000 people. That ...
(Date:10/20/2014)... The newly updated Worldwide Medical Market Forecasts to ... figures and forecasts on the global medical markets. , ... the Worldwide Medical Market Forecasts to 2019 ... the worldwide medical market to reveal key insights into ... for 2010-2019, covering each country market individually, allowing you ...
Breaking Medicine News(10 mins):Health News:HealthGuideMD Presents Ways to Improve Your Exercise and Fitness 2Health News:Akeso Biomedical Appoints Dr. Anthony J. Sinskey to Board of Directors 2Health News:Teen Sisters Develop Ways to Measure Lung, Heart Damage 2Health News:Tire Company Sets Standard for Ebola Care in Liberia: CDC 2Health News:Worldwide Medical Market Forecasts to 2019 a New Research Report Added to ReportsnReports.com 2Health News:Worldwide Medical Market Forecasts to 2019 a New Research Report Added to ReportsnReports.com 3Health News:Worldwide Medical Market Forecasts to 2019 a New Research Report Added to ReportsnReports.com 4
... of cancer. Cells without this protein are usually rendered genetically ... it from growing. However sometimes mutations alter cells in such ... Researchers indicated that loss of E2F3 itself doesn't cause cancer ... change into a cancer cell.// E2F3 is part of a ...
... indicates that Negative exercise electrocardiograph tests should ... of hypertensive patients with chest pain. Even ... risk, positive findings could not distinguish levels ... pharmacological stress echocardiography yielded diagnostic and prognostic ...
... SARS patients in India went up to 10, authorities are ... India has shown a far higher recovery rate than the ... and superior medical care provided to SARS patients. Even what ... and they are only between 60 and 65 per cent ...
... With a severe blow by SARS, the economies of ... quarter. More than five million foreign workers, from Filipina ... the wheels of commerce in the region, sending home ... families. Studies show most of those families used the ...
... victims have been refused treatment in Indian clinics where ... have been moved to government hospital. Private medical centre ... SARS infected patients. The patients include a bride, her ... the ceremony from Indonesia via Singapore. There were also ...
... SARS on a plane trip may probably be small if ... too little is currently known about the virus. However people ... strong filters against viruses. There have even been policies implemented ... equipped with sophisticated air cleaning systems that has stringent filters ...
Cached Medicine News:
(Date:10/22/2014)... AUSTIN, Texas , Oct. 22, 2014 /PRNewswire/ ... they have entered into a license and collaboration ... target cancers. PhosImmune possesses technology for ... surfaces of tumor cells in association with HLA ... proteins that play a role in malignancy, are ...
(Date:10/22/2014)... , Oct. 22, 2014  Regulus ... a biopharmaceutical company leading the discovery and ... announced that it has demonstrated human proof-of-concept ... clinical study evaluating RG-101, a wholly-owned, GalNac-conjugated ... of hepatitis C virus infection ("HCV").  Interim ...
(Date:10/20/2014)... BOSTON , Oct. 20, 2014  Indianapolis ... and Toronto -based 3D visualization ... the Congress of Neurological Surgeons Annual Meeting that ... technologies for brain surgery. The collaboration is the ... powerful combination to improve patient outcomes. NICO,s BrainPath® ...
Breaking Medicine Technology:Biotech Companies To Co-Discover And Co-Develop Antibodies Targeting Novel Phosphopeptide Tumor Targets 2A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 2A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 3A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 4A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 5A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 6A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 7A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 8Indianapolis' NICO Corporation and Toronto's Synaptive Medical, Inc. Announce First-Ever Intervention-Visualization Partnership with Goal to Improve Standard of Care in Neurosurgery 2Indianapolis' NICO Corporation and Toronto's Synaptive Medical, Inc. Announce First-Ever Intervention-Visualization Partnership with Goal to Improve Standard of Care in Neurosurgery 3Indianapolis' NICO Corporation and Toronto's Synaptive Medical, Inc. Announce First-Ever Intervention-Visualization Partnership with Goal to Improve Standard of Care in Neurosurgery 4Indianapolis' NICO Corporation and Toronto's Synaptive Medical, Inc. Announce First-Ever Intervention-Visualization Partnership with Goal to Improve Standard of Care in Neurosurgery 5
... and INDIANAPOLIS , March 15 ... evaluated response rates in patients with a common genetic ... TRITON-TIMI 38 study were treated with dual antiplatelet therapy ... plus aspirin and managed with percutaneous coronary intervention (PCI) ...
... March 15 At this year,s annual meeting ... 14-16 in Atlanta , Royal Philips Electronics ... simplifying cardiology patient care by highlighting a range of ... tools, intuitive information management systems, new temperature modulation therapies, ...
Cached Medicine Technology:New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 2New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 3New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 4New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 5New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 6New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 7New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 8New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 9New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 10New Analysis Assesses Impact of Common Genetic Variation on Benefit of Antiplatelet Therapy 11Philips Simplifies Cardiology Care With Technologies That Span the Entire Spectrum of Patient Care 2Philips Simplifies Cardiology Care With Technologies That Span the Entire Spectrum of Patient Care 3Philips Simplifies Cardiology Care With Technologies That Span the Entire Spectrum of Patient Care 4Philips Simplifies Cardiology Care With Technologies That Span the Entire Spectrum of Patient Care 5Philips Simplifies Cardiology Care With Technologies That Span the Entire Spectrum of Patient Care 6Philips Simplifies Cardiology Care With Technologies That Span the Entire Spectrum of Patient Care 7Philips Simplifies Cardiology Care With Technologies That Span the Entire Spectrum of Patient Care 8Philips Simplifies Cardiology Care With Technologies That Span the Entire Spectrum of Patient Care 9
... today's busy physician's office environment, ... contribute to timing errors and ... strips are read visually. Studies ... is performed with an instrument, ...
Keep your laboratory efficient. And meet your volume needs. When you set the pace with the Clinitek 500 Urine Chemistry Analyzer. Its ready when you are. No warm-up. No calibration. No bells. Just pr...
... pressure sensor catheters incorporate a ... injection/guide wire, or high-speed injection/guide ... high-speed injection has two laterally ... an open-end pigtail extension for ...
... catheters provide a simple, efficient and ... in the heart or cardiovascular system. ... ideal for high fidelity pressure measurement. ... 2 to 7 French and in ...
Medicine Products: