Navigation Links
Amicus Therapeutics Presents Preclinical Data from Studies of,Plicera for Gaucher Disease

Studies Show Increased Enzyme Levels in Two Most Common Mutations

CRANBURY, N.J., March 20, 2007 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of a range of human genetic diseases, announced today that it will present the results of preclinical studies of Plicera(TM) (isofagomine tartrate, AT2101) for Gaucher disease at the of Medical Genetics (ACMG) annual meeting March 21-25 in Nashville, TN. The data demonstrate the ability of Plicera to increase levels of the target enzyme in cells derived from a patient with the N370S mutation and in mice that express the L444P mutation. The N370S and the L444P are the two most common mutations associated with Gaucher disease. Additional data from both studies will also be presented.

Plicera is designed to selectively bind to and stabilize GCase, the enzyme deficient in Gaucher disease. This deficiency leads to lysosomal accumulation of glucocerebroside inside certain cells, which is believed to cause the various symptoms of Gaucher disease. Plicera facilitates proper trafficking of the enzyme to the lysosomes, the compartments in the cell where it is needed to break down glucocerebroside.

The following is a summary of the preclinical Plicera data being presented at ACMG.

    - In vitro exposure to Plicera increased transport of GCase to the

      lysosomes in cells derived from a patient with the N370S mutation.

      Once in the lysosome, the enzyme was stable and active for more than 3

      days after Plicera was removed. The N370S is the most common mutation

      associated with Gaucher disease in the Western world.  These studies

      were published in the September 12, 2006 issue of the Proceedings of the

      National Academy of Sciences (PNAS).


    - Oral administration of Plicera resulted in a dose-dependent increase of
'"/>




Page: 1 2 3

Related medicine technology :

1. Amicus Therapeutics Presents Positive Results From Phase 1 Clinical Studies of Plicera for Gaucher Disease
2. Amicus Therapeutics Presents Data From Preclinical and Phase 1 Studies of Amigal for Fabry Disease
3. Cell Therapeutics, Inc. Management to Discuss Todays Announcement of Interim Pixantrone Study Results
4. Halozyme Therapeutics Enhanze Technology Large Protein Molecule Therapeutic Clinical Trial Results Presented at the 34th Annual Meeting of the Controlled Release Society
5. CV Therapeutics Identifies Potential Anti-Diabetic Mechanism of Action for Ranexa
6. Fat Kills Cancer: Turning Stem Cells from Fat Tissue into Personalized, Cancer-Targeted Therapeutics
7. A 4-Week Therapy with Transition Therapeutics E1-I.N.T. Leads to Sustained Reductions in Blood Glucose Levels for 6 Months Post-treatment in Type 2 Diabetes Patients
8. Clinical Trial Suggests Bone Marrow Stem Cells Are Useful for Spinal Cord Injury; PrimeCell Therapeutics Provided Pre-Clinical Study
9. Opexa Therapeutics Reports Positive Top-line Data in Phase I/II Extension Trial with Tovaxin for Multiple Sclerosis
10. Orexigen Therapeutics to Present Data on Contrave Impact on Visceral Fat and Insulin Resistance at American Diabetes Association 67th Scientific Sessions in Chicago June 23
11. Allos Therapeutics Reports Results for Phase 3 Enrich Study of Efaproxyn in Women With Brain Metastases Originating From Breast Cancer
Post Your Comments:
(Date:1/15/2014)... , Jan. 15, 2014 Reportlinker.com ... report is available in its catalogue: ... and Technologies for Advanced Drug Delivery ... Administration http://www.reportlinker.com/p01941928/Global-Markets-and-Technologies-for-Advanced-Drug-Delivery-Systems----Focus-on-Routes-of-Administration.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Drug_Delivery_Technology ...
(Date:1/14/2014)... 2014 InformEx, North America,s leading ... will hold the 30 th edition of its annual ... Center from January 21-24. (Logo: http://photos.prnewswire.com/prnh/20131028/NY05330LOGO-a ) ... fine chemical and specialty chemical companies will take part in ...
(Date:1/14/2014)... 14, 2014   NuAire , a manufacturer of ergonomically designed ... Hitachi Koki of Japan to sell ... America . NuAire will utilize its network of sales professionals ... Canada to offer assistance in application use, product benefits ...
Breaking Medicine Technology:Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 2Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 3Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 4Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 5Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 6Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 7Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 8Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 9Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 10Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 11Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 12Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 13Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 14Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 15Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on Routes of Administration 16Media Advisory: InformEx 2014 Events & Speaker Lineup for 30th Edition Conference in Miami Beach 2NuAire announces sales and service of Hitachi Koki centrifuges in North America 2
... The Child Neurology Foundation (CNF) today announced the launch of ... The website, www.infantilespasmsinfo.org , is an educational ... components of Infantile Spasms Awareness Week. , Earlier ... Awareness Week will place this week in conjunction with the ...
... , WASHINGTON, Oct. 12 Today at the ... #219), leading provider of Good Promotional Practices software, ... in integrated hotline and case management solutions. Together, ... compliance monitoring to help Life Sciences companies cope ...
Cached Medicine Technology:Child Neurology Foundation Announces the Launch of Infantilespasmsinfo.org 2Child Neurology Foundation Announces the Launch of Infantilespasmsinfo.org 3Prolifiq and EthicsPoint Announce Partnership: 'Automatic Alerts' Help Life Sciences Engage in Good Promotional Practices 2Prolifiq and EthicsPoint Announce Partnership: 'Automatic Alerts' Help Life Sciences Engage in Good Promotional Practices 3
(Date:4/18/2014)... scientists could lead to potential new treatments for breaking ... , Fibrosis, or scarring, is a hallmark of ... lungs can lead to serious organ damage and, in ... options centers on findings made by Swati Bhattacharyya, PhD, ... that a specific protein plays in promoting fibrosis. ...
(Date:4/17/2014)... variant specifically associated with the risk of a ... cent of all breast cancer cases. , The ... called invasive lobular carcinoma, gives researchers important clues ... of breast cancer, which can be missed through ... the journal PloS Genetics , was co-led ...
(Date:4/17/2014)... PLOS Pathogens , children who live in regions of ... immune response to infection with malaria parasites that may ... and illness and partially control the growth of malaria ... develop future interventions that prevent or mitigate the disease ... 200 million cases of malaria occur worldwide, resulting in ...
(Date:4/17/2014)... 2014) Two recent papers by a University ... may help scientists develop treatments or vaccines for ... encephalitis and other disease-causing flaviviruses. , Jeffrey S. ... genetics at the School of Medicine and an ... Institute, and colleagues recently published articles in the ...
(Date:4/17/2014)... team has created a new technology for modifying ... travel the body and selectively target cancer and ... devices that monitor and modify human physiology is ... no existing technology enabled bioengineers to build such ... respond in a customized fashion. , "The project ...
Breaking Medicine News(10 mins):Health News:New clues on tissue scarring in scleroderma 2Health News:First genetic link discovered to difficult-to-diagnose breast cancer sub-type 2Health News:First genetic link discovered to difficult-to-diagnose breast cancer sub-type 3Health News:Study sheds light on how the immune system protects children from malaria 2Health News:CU researchers discover target for treating dengue fever 2Health News:Building 'smart' cell-based therapies 2
... ... ... ... ...
... ... , ... , , , ... NEW YORK , March 4 ...
... ... ... ... ...
... third leading cause of cancer death among men and ... CRC screening receive it. The American Gastroenterological Association (AGA) ... of screening and to encourage everyone age 50 and ... that those without insurance have limited access to screening, ...
... Calculus Bovis compound preparation (ICCBco) in the treatment ... schistosomiasis as the experimental animal model, a research ... trial to observe pathological changes and pathological effect ... the lung tissue of portal hypertensive rabbits with ...
... at risk, but elderly women are too, study finds , ... common cause of shoulder dislocation, and young males have the ... study shows. , Researchers studied 8,940 shoulder dislocations treated at ... to 2006 and found that the overall incidence rate was ...
Cached Medicine News:Health News:'Less Is More' with the Innovation of Avemar(R) Fermented Wheat Germ Extract 2Health News:'Less Is More' with the Innovation of Avemar(R) Fermented Wheat Germ Extract 3Health News:'Less Is More' with the Innovation of Avemar(R) Fermented Wheat Germ Extract 4Health News:'Less Is More' with the Innovation of Avemar(R) Fermented Wheat Germ Extract 5Health News:Cowen Group Announces Promotions in Health Care Investment Banking 2Health News:Cowen Group Announces Promotions in Health Care Investment Banking 3Health News:Alan Henry, M.D. Joins MedSolutions as Chief Medical Officer of Premerus 2Health News:Alan Henry, M.D. Joins MedSolutions as Chief Medical Officer of Premerus 3Health News:Alan Henry, M.D. Joins MedSolutions as Chief Medical Officer of Premerus 4Health News:Alan Henry, M.D. Joins MedSolutions as Chief Medical Officer of Premerus 5Health News:Alan Henry, M.D. Joins MedSolutions as Chief Medical Officer of Premerus 6Health News:Physicians come together on National Colorectal Cancer Awareness and Screening Day 2Health News:Treatment of portal hypertensive pulmonary lesions induced by schistosomiasis 2Health News:Shoulder Dislocations a Sports Hazard 2
... and Collections Administrator has a maximum ... models and personalized paper or electronic ... ability to facilitate rebilling, ledger notes, ... from the Onscreen Collections Worksheet and ...
Visionary OFFICE™ PM offers a Windows interface featuring medical billing, appointment scheduling and patient information....
A.I.med's medical billing software is a medical billing software. The billing module integrates with A.I.med's Electronic Medical Record software. A.I.med's medical billing module also integrates wit...
... The OmniMD Medical Billing system allows ... and accounting. It is integrated with ... capture. The system interfaces with electronic ... tracking. With a set of reports, ...
Medicine Products: