Navigation Links
Amicus Therapeutics Presents Positive Results From Phase 1 Clinical,Studies of Plicera for Gaucher Disease

ent increase in GCase levels was observed in white blood cells from healthy volunteers receiving Plicera.

Phase 2 Clinical Trials of Plicera in Gaucher Disease

Based on the Phase 1 results, Amicus has initiated two Phase 2 clinical trials of Plicera for Gaucher disease. One is a 4-week study designed to evaluate the safety and pharmacodynamic effects of Plicera in Type I Gaucher patients who will discontinue enzyme replacement therapy for the duration of the study. The second is a 6-month study designed to evaluate the safety of Plicera and its effect on parameters that are commonly abnormal in Gaucher disease. This study will be conducted in Type I Gaucher patients who have never received enzyme replacement therapy. More information regarding these studies can be found at www.clinicaltrials.gov and www.amicustherapeutics.com.

About Gaucher Disease

Gaucher disease, the most commonly diagnosed lysosomal storage disorder, is caused by inherited genetic mutations in the GBA gene, which result in deficient activity of the enzyme acid beta-glucosidase, also known as glucocerebrosidase (GCase). Deficient GCase activity leads to lysosomal accumulation of glucocerebroside inside certain cells, which is believed to cause the various symptoms of Gaucher disease, including an enlarged liver and spleen, abnormally low levels of red blood cells and platelets and skeletal complications. In some cases there is significant impairment of the central nervous system. Gaucher disease affects an estimated 8,000 to 10,000 people worldwide. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for the active ingredient in Plicera in the United States.

About Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company developing novel, oral therapeutics
'"/>




Page: 1 2 3

Related medicine technology :

1. Amicus Therapeutics Presents Preclinical Data from 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:7/31/2015)... According to a new market research report " ... Oxide, Filtration, E-beam, Gamma), Services (Onsite & Offsite), Consumables (Indicators, ... to 2020", published by MarketsandMarkets, The Sterilization Equipment Market is ... a CAGR of 7.6%.from 2015 to 2020. ... ables and 37 F igures ...
(Date:7/31/2015)... Inc. (Nasdaq: SMLR ), a company that provides ... groups, today reported financial results for the second quarter ... second quarter of 2015, Semler reported continued progress with ... quarter revenue growth of 8%, and quarter over quarter ... 18%," said Doug Murphy-Chutorian, M.D., chief executive officer of ...
(Date:7/31/2015)... Magnaquest, a leading Subscription ... cloud computing operators has signed a Proof of ... Internet of Things (IoT) related initiative. The PoC ... model.      (Logo: http://photos.prnewswire.com/prnh/20140807/700645 ) ... business innovations to make healthcare services more accessible ...
Breaking Medicine Technology:Sterilization Equipment Market is Expected to Reach USD 6.1 Billion by 2020 2Sterilization Equipment Market is Expected to Reach USD 6.1 Billion by 2020 3Sterilization Equipment Market is Expected to Reach USD 6.1 Billion by 2020 4Semler Reports Second Quarter 2015 Financial Results 2Semler Reports Second Quarter 2015 Financial Results 3Semler Reports Second Quarter 2015 Financial Results 4Semler Reports Second Quarter 2015 Financial Results 5Semler Reports Second Quarter 2015 Financial Results 6Magnaquest Signs a POC With a Leading Pharma Giant in Europe to Manage Subscription Management and Billing for Internet of Things (IoT) 2
(Date:7/31/2015)... ... July 31, 2015 , ... Jacksonville-based drug and ... True North Conference Center. The medical community, social workers, law enforcement, and government ... more aligned in the effort to better understand and combat sex trafficking. Lead ...
(Date:7/31/2015)... (PRWEB) , ... July 31, 2015 , ... M3 USA ... excited to launch three new Board Exam Courses. , The additions of Gastroenterology, ... Board Exam Prep from MDLinx makes preparing for the exam easy and painless. ...
(Date:7/31/2015)... ... July 31, 2015 , ... According to an article published ... if they could reach a consensus on the steps that need to be taken ... group reached 90 percent consensus on a series of steps that each surgeon must ...
(Date:7/31/2015)... (PRWEB) , ... July 31, 2015 , ... ... educating both readers and potential patients alike on various plastic surgery procedures including, ... covered also include such popular dermatology procedures like Botox and fillers. , ...
(Date:7/31/2015)... (PRWEB) , ... July 31, 2015 , ... Transfinder, a ... 2015. , Total sales were up 16 percent year-over-year for the first six months ... Antonio Civitella said he expected Transfinder to beat last year’s record-revenue mark of $10.4 ...
Breaking Medicine News(10 mins):Health News:Lakeview Health Links Sex Trafficking and Addiction in Special Jacksonville Educational Workshop 2Health News:Lakeview Health Links Sex Trafficking and Addiction in Special Jacksonville Educational Workshop 3Health News:MDLinx Offers Three New Board Exam Prep Courses - Gastroenterology, Pulmonology and Allergy/Immunology 2Health News:MDLinx Offers Three New Board Exam Prep Courses - Gastroenterology, Pulmonology and Allergy/Immunology 3Health News:Article on Plastic Surgery Consensus Highlights the Duties of Veteran Plastic Surgeons, says Beverly Hills Physicians 2Health News:Article on Plastic Surgery Consensus Highlights the Duties of Veteran Plastic Surgeons, says Beverly Hills Physicians 3Health News:Cosmetic Town: Now Providing Interactive And Vital Medical Knowledge 2Health News:Transfinder Closes First Half of 2015 with Record Revenue 2
... Awareness of genetic testing was higher among adults in ... use of genetic testing was lower, according to data ... of Cancer Health Disparities, held Sept. 30 to Oct ... direct-to-consumers through the Internet and other venues, providing individual ...
... radiology department at a mid-sized hospital were able to ... method for measuring general technologist productivity, according to a ... the American College of Radiology ( www.jacr.org ). ... competing priorities that may be difficult to achieve simultaneously," ...
... have discovered that the over-activation of a single protein ... and facilitate the most common form of Parkinson,s disease. ... new targets for drugs already known to inhibit it, ... 1 million older Americans. Previous research demonstrated ...
... lung CT scans to improve treatment of lung cancer ... a physician-scientist at The University of Texas MD Anderson ... Institutes of Health. The award, one of 52 ... over five years to Thomas Guerrero, M.D., Ph.D., associate ...
... review article published in the October issue of ... Clinic physicians differentiate the ethical and legal permissibility ... comfort measures, specifically palliative sedation, from that of ... that palliative sedation has an important place on ...
... aboard NASA,s James Webb Space Telescope is getting its first ... Appleton Laboratory (RAL) in the United Kingdom. The Mid-InfraRed Instrument ... as diverse as the first light in the early Universe ... "The start of space simulation testing of the MIRI is ...
Cached Medicine News:Health News:Adults in Puerto Rico aware of genetic testing, but use remains low 2Health News:Parkinson's disease: Excess of special protein identified as key to symptoms and possible new target for treatment with widely used anti-cancer drug imatinib 2Health News:Parkinson's disease: Excess of special protein identified as key to symptoms and possible new target for treatment with widely used anti-cancer drug imatinib 3Health News:NIH award goes to UT MD Anderson imaging innovator Guerrero 2Health News:NIH award goes to UT MD Anderson imaging innovator Guerrero 3Health News:NASA's Webb telescope MIRI instrument takes one step closer to space 2Health News:NASA's Webb telescope MIRI instrument takes one step closer to space 3Health News:NASA's Webb telescope MIRI instrument takes one step closer to space 4
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
... a national reference laboratory and a worldwide ... ARUP offers an extensive test menu of ... clinical and anatomic pathology. Owned by the ... more than half of the nation's university ...
... ARUP Laboratories is a national reference ... laboratory research and development. ARUP offers an ... unique medical tests in clinical and anatomic ... ARUP Laboratories' clients include more than half ...
... a national reference laboratory and a worldwide ... ARUP offers an extensive test menu of ... clinical and anatomic pathology. Owned by the ... more than half of the nation's university ...
Medicine Products: