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:7/29/2014)... Petersburg, FL (PRWEB) July 30, 2014 Phytceramides are ... how and why in her new book. , Today is the ... Phytoceramides: Anti Aging At Its Best . The book is also ... skin care book, but felt that Phytoceramides were so ... to bring out this focused book now," says Kathy Heshelow, founder ...
(Date:7/29/2014)... Dublin, Ireland (PRWEB) July 30, 2014 ... announced the successful completion of a Phase I trial ... Acid (DGLA). , This study was a randomized, placebo-controlled, ... to assess the safety, pharmacokinetics and effect of food ... confirmed the benign safety profile of DGLA following both ...
(Date:7/29/2014)... July 29, 2014 The Mesothelioma ... the Deadliest Cancer Coalition, led advocacy efforts among ... the newly established Congressional Caucus on the Deadliest Cancers. ... caucus now features the following 20 members: , Rep. Matt ... – May 2, 2014, Rep. Zoe Lofgren (CA-19) – June ...
(Date:7/29/2014)... they have discovered a chemical alteration in a single ... in larger studies, could give doctors a simple blood ... suicide. , The discovery, described online in The ... a gene involved in the function of the brain,s ... turning what might otherwise be an unremarkable reaction to ...
(Date:7/29/2014)... rich in soy may help feminine hearts, but timing ... Menopause , the journal of The North American ... diet of women in Asia, produces the least atherosclerosis. ... Asian migrants to North America, leads to just as ... to soy from a Western diet after menopause helps ...
Breaking Medicine News(10 mins):Health News:Last Day New Book Is Available at No Cost: Phytoceramides, Anti Aging At Its Best by Sublime Beauty Founder® Kathy Heshelow 2Health News:Last Day New Book Is Available at No Cost: Phytoceramides, Anti Aging At Its Best by Sublime Beauty Founder® Kathy Heshelow 3Health News:DS107G-01 – Successful Completion of Phase I Oral DGLA Clinical Trial 2Health News:Mesothelioma Organization Works to Add Members to ‘Deadliest Cancer Caucus’ 2Health News:Mesothelioma Organization Works to Add Members to ‘Deadliest Cancer Caucus’ 3Health News:A blood test for suicide? 2Health News:A blood test for suicide? 3Health News:Soy may help women's hearts if they start early 2
... -- A new study finds that many American children aged ... they ride in cars -- a practice that could prove ... National Highway Traffic Safety Administration survey of nearly 900 drivers. ... 9-year-old passengers use seat belts and more than three-quarters of ...
... FRIDAY, Nov. 18 (HealthDay News) -- Although one in four ... of colon surgery, new research reveals these readmissions -- which ... The most common reason colorectal surgery patients return to ... published in the December issue of the journal Diseases ...
... computing system broke new ground earlier this year when it ... Now, The Scripps Research Institute is hoping to do something ... of the prize money from that tournament: Find a cure ... To that end, Scripps Research and IBM (NYSE: ...
... Nov. 18 (HealthDay News) -- A "tongue-tie" condition in infants ... many cases, an expert says. The condition, also known ... newborns. The most common type is easy to identify and ... tongue indentation at the front of the mouth. Another ...
... HealthDay Reporter , FRIDAY, Nov. 18 (HealthDay News) -- Those ... foods around. U.S. government health experts now encourage ... Weight Watchers, new system assigns no points to nearly all ... But, really, what,s the big deal? Why is it so ...
... rates due to colorectal cancer have declined overall, yet ... suggests that underuse of colonoscopy screening among these groups ... New York City launched a multifaceted campaign to improve ... A new study conducted by researchers at Columbia University,s ...
Cached Medicine News:Health News:Ill-Fitting Children's Seat Belts Common in U.S., Study Finds 2Health News:Colon Surgery Linked to High Readmission Rates 2Health News:Jeopardy! winnings spur IBM and Scripps Research Institute collaboration to fight against malaria 2Health News:Jeopardy! winnings spur IBM and Scripps Research Institute collaboration to fight against malaria 3Health News:Easy-to-Treat Condition Can Disrupt Breast-Feeding 2Health News:For Nutritional Punch, You Can't Beat Plant-Based Foods 2Health News:For Nutritional Punch, You Can't Beat Plant-Based Foods 3Health News:For Nutritional Punch, You Can't Beat Plant-Based Foods 4Health News:Colon cancer screening campaign erases racial, gender gaps in use of colonoscopy 2Health News:Colon cancer screening campaign erases racial, gender gaps in use of colonoscopy 3
(Date:7/30/2014)... California , July 30, 2014 ... technology and CUDA programming expertise to big data, ... markets  eInfochips, a leading engineering R&D ... clients to develop NVIDIA GPU-powered solutions for ... high-performance computing (HPC), industrial , ...
(Date:7/30/2014)... 2014 Die Apex Medical Corporation ... Bereich Atemtherapie- und Druckzonenprodukte, gibt bekannt, dass ... Deutschland anfechten werden. Dieses hat entschieden, dass ... von ResMed verletze. Apex weist darauf hin, ... verschiedene Patente von ResMed Anfechtungen wegen Nichtigkeit ...
(Date:7/29/2014)... Israel , July 30, 2014  Immune ... Stockholm : "IMNP."; "Immune" or "the ... of patients for a Phase II proof of concept ... in the treatment of moderate to severe bullous pemphigoid. ... receive two bertilimumab infusions, at a dose of 10 ...
Breaking Medicine Technology:eInfochips to Develop NVIDIA GPU-powered Solutions for Aerospace and Defense, Medical Imaging, HPC and Visual Computing 2Apex setzt seinen Kampf gegen Patente für einen freien Wettbewerb fort 2Apex setzt seinen Kampf gegen Patente für einen freien Wettbewerb fort 3Immune Pharmaceuticals Commences Patient Screening For Bertilimumab Phase II Clinical Trial In Bullous Pemphigoid, A Rare Auto-Immune Disease Of The Skin 2Immune Pharmaceuticals Commences Patient Screening For Bertilimumab Phase II Clinical Trial In Bullous Pemphigoid, A Rare Auto-Immune Disease Of The Skin 3Immune Pharmaceuticals Commences Patient Screening For Bertilimumab Phase II Clinical Trial In Bullous Pemphigoid, A Rare Auto-Immune Disease Of The Skin 4Immune Pharmaceuticals Commences Patient Screening For Bertilimumab Phase II Clinical Trial In Bullous Pemphigoid, A Rare Auto-Immune Disease Of The Skin 5
... is little evidence to support the use of atypical ... approved purposes, even though many clinicians continue to commonly ... to a new report from the U.S. Department of ... and Quality (AHRQ). An article based on information in ...
... The Houston Business Journal recently announced that eCardio Diagnostics ... Fast 100, which included a distinguished list of 100 of ... eCardio was ranked No. 17 with a two-year growth ... to receive this prestigious award. "There were so ...
Cached Medicine Technology:"Off-Label" Use of Antipsychotic Drugs for Some Conditions Not Supported By Evidence 2eCardio Among Honorees of the Houston Business Journal's Fast 100 List for Second Consecutive Year 2
For use with E5381 600 Circling Band (40 style). 2.2 mm diameter, 28.5 mm long....
Used with Double Tantalum Clip (250 style) E5381 852. Overall length: 7.0 mm....
Concave inner surface to conform to globe. Used in conjunction with the E5381 700 Circling Band (240 style). Indications: wide scleral bed. Outside diameter 31.7 mm....
Used in conjunction with the E5381 700 Circling Band (240 style). Indications: Narrow scleral bed. Overall length: 100 mm, 3.9 inches....
Medicine Products: