Navigation Links
Amicus Therapeutics Announces Positive Results From Two Phase 1 Clinical Studies of AT2220 for Pompe Disease
Date:10/23/2007

CRANBURY, N.J., Oct. 23 /PRNewswire-FirstCall/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of a range of human genetic diseases, announced today positive results from two recently completed Phase 1 clinical studies of AT2220 (1-deoxynojirimycin HCl) for Pompe disease. The Phase 1 results show that AT2220 was well-tolerated in healthy volunteers with good oral bioavailability and pharmacokinetic parameters. These results will be presented at the American Society of Human Genetics (ASHG) Annual Meeting on October 23-27 in San Diego, CA.

AT2220 is designed to selectively bind to and stabilize acid a-glucosidase (GAA), the enzyme deficient in Pompe disease. This deficiency leads to lysosomal accumulation of glycogen inside muscle cells, which is believed to cause the various symptoms of Pompe disease. AT2220 facilitates proper trafficking of the enzyme to the lysosomes, the compartments in the cell where it is needed to break down glycogen. AT2220 has been shown to increase GAA levels in cell lines derived from Pompe patients, in transfected cells expressing mutant forms of GAA, and in healthy mice and monkeys.

Two double-blind, placebo-controlled, dose escalation Phase 1 studies in healthy volunteers were completed. These studies were designed to evaluate the safety, tolerability and pharmacokinetics of AT2220. In a single ascending dose study, 32 individuals received oral doses of 50, 150, 300, or 600 mg AT2220 or placebo. In a multiple ascending dose study, 24 individuals received oral doses of 50, 150, or 450 mg/day AT2220 or placebo for 7 days. In both studies, AT2220 was generally safe and well-tolerated at all doses and was orally bioavailable with a plasma half-life of 4 to 5 hours. There were no drug-related serious adverse events and no adverse events were considered to be definitely or probably related to study treatment. In the multiple asce
'/>"/>

SOURCE Amicus Therapeutics
Copyright©2007 PR Newswire.
All rights reserved

Page: 1 2 3 4

Related medicine technology :

1. Amicus Therapeutics Presents Positive Results From Phase 1 Clinical Studies of Plicera for Gaucher Disease
2. Amicus Therapeutics Presents Preclinical Data from Studies of Plicera for Gaucher Disease
3. Amicus Therapeutics Presents Data From Preclinical and Phase 1 Studies of Amigal for Fabry Disease
4. Cell Therapeutics, Inc. Management to Discuss Todays Announcement of Interim Pixantrone Study Results
5. Halozyme Therapeutics Enhanze Technology Large Protein Molecule Therapeutic Clinical Trial Results Presented at the 34th Annual Meeting of the Controlled Release Society
6. CV Therapeutics Identifies Potential Anti-Diabetic Mechanism of Action for Ranexa
7. GTC Biotherapeutics to Webcast Corporate Presentation at the C.E. Unterberg, Towbin Emerging Growth Life Opportunities Conference
8. Fat Kills Cancer: Turning Stem Cells from Fat Tissue into Personalized, Cancer-Targeted Therapeutics
9. 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
10. Clinical Trial Suggests Bone Marrow Stem Cells Are Useful for Spinal Cord Injury; PrimeCell Therapeutics Provided Pre-Clinical Study
11. Pieris Progresses Development of its Proprietary Biotherapeutics Platform: Successful Demonstration of Dual Targeting With Duocalin(R) Technology
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/17/2014)... Sept. 17, 2014 Brazil ... Summary GlobalData,s new report, "Brazil ... market data on the Brazil Gynecological Devices market. ... dollars, volume (in units) and average prices (in ... Devices (Transcervical Sterilization Devices (Micro-inserts) and Mechanical Sterilization ...
(Date:9/17/2014)... Sept. 17, 2014  Levi & Korsinsky, LLP is ... possible claims of breaches of fiduciary duty by the ... get more information, click here: http://zlk.9nl.com/alexza-pharmaceuticals-alxa . There ... If you own common stock in Alexza Pharmaceuticals Inc. ... Eduard Korsinsky, Esq. either via email at ...
(Date:9/17/2014)... PITTSBURGH , Sept. 17, 2014 /PRNewswire-iReach/ ... a white paper discussing improvements in the ... anatomic structures for both normal and diseased ... real-time information, simplifies surgical procedures, lowers surgical ... robot-assisted surgical procedures. The ...
Breaking Medicine Technology:Brazil Gynecological Devices Market Outlook to 2020 2Brazil Gynecological Devices Market Outlook to 2020 3Patient Outcomes Improve When Molecular Chemical Imaging Technology Is Used to Aid Robotic Surgical Procedures, According to New White Paper 2
... YORK, April 20, 2011 Reportlinker.com announces that a ... Successful Strategies for Drug Repositioning ... Introduction This report Highlights ... to drug development. The success of the approach is ...
... Sagent Pharmaceuticals (Nasdaq: SGNT ) announced the ... of the offering has been increased from the previously announced ... stock at a price to the public of $16.00 per ... NASDAQ Global Market on April 20, 2011 under the ticker ...
Cached Medicine Technology:Reportlinker Adds Successful Strategies for Drug Repositioning 2Reportlinker Adds Successful Strategies for Drug Repositioning 3Reportlinker Adds Successful Strategies for Drug Repositioning 4Sagent Pharmaceuticals Upsizes and Prices Initial Public Offering 2
(Date:9/17/2014)... 17, 2014 As kinship care continues ... who cannot live with their parents, emphasis on kinship ... to enhance services and supports for kinship caregivers and ... National Kinship Care Conference is being held this week ... best practice, policy, program and service development for kinship ...
(Date:9/17/2014)... infection may be preventable with a vaccine, new research ... researchers at Washington University School of Medicine in St. ... tubes used in hospitals and other care facilities to ... day a catheter is present in the urethra and ... Nearly every patient who has a catheter for more ...
(Date:9/17/2014)... News) -- Social networking sites can help people lose ... who are obese, sites like Twitter and Facebook can ... doctors as well as peers, researchers found. "One ... that it offers the potential to be much more ... to traditional approaches," the study,s lead author, Dr. Hutan ...
(Date:9/17/2014)... 2014 In Pretrial Order #139 (Scheduling ... discovery conferences would be beneficial in this [MDL].” Accordingly, ... “At the end of December,” the order further provides, ... additional conferences. , “The plain import of this ... still much to be discussed as these cases progress ...
(Date:9/17/2014)... News) -- Positive messages about the health benefits of ... a new study suggests. Although smokers who think ... about the harmful effects of smoking, researchers found smokers ... more from "gain-framed," or positive, messages about how quitting ... using a mix of both types of messages might ...
Breaking Medicine News(10 mins):Health News:Arizona’s Children Association Kinship Service Providers Invited to Present at the CWLA National Conference 2Health News:In mice, vaccine stops urinary tract infections linked to catheters 2Health News:In mice, vaccine stops urinary tract infections linked to catheters 3Health News:Social Networking Sites Can Help Obese Lose Weight: Study 2Health News:Transvaginal Mesh Lawsuit News: New Order in C.R. Bard MDL Sets Schedule of Conferences for September to December, the Rottenstein Law Group LLP Reports 2Health News:More Than One Kind of Message May Convince Smokers to Quit, Study Says 2
... A newly published report reveals patients with epilepsy and ... uncontrolled seizures, drug-related side effects, and a lower overall ... SES patients used the hospital emergency room more often ... epileptic patients at higher socioeconomic levels. Full findings are ...
... be a helpless and heartbreaking situation for families as they ... What they may not know is that there,s a society ... from this dangerous and possibly fatal behavior. University ... of social support group as social networks grow on the ...
... Hospital of Rhode Island has been named a Center ... Institute of OptumHealth, an independent audit company. According ... equal. For complex medical conditions, the disparity is ... developed Centers of Excellence networks that provide access to ...
... some 19th century Mormon men had the curious effect of ... marriages, say scientists from Indiana University Bloomington and three other ... Human Behavior . Simply put, the more sister-wives a ... produce. "Although it,s great in terms of number of ...
... to trauma patients, according to a study published in the ... have death rates that are 34 percent lower, while spending ... hospitals, suggesting high quality can coexist with lower cost. ... The research comes at a time when the affordability of ...
... HealthDay Reporter , MONDAY, Feb. 21 (HealthDay News) ... heavily on the emotional lives of women than men, a ... imply that women need additional care, including follow-up and possibly ... by Dr. Cynthia Holterhues of the department of dermatology ...
Cached Medicine News:Health News:Health care disparities seen in epilepsy patients with low socioeconomic status 2Health News:Research uncovers a secret society connecting through the Internet 2Health News:Women & Infants named Center of Excellence for Neonatal Care for fifth year 2Health News:Polygamy hurt 19th century Mormon wives' evolutionary fitness 2Health News:Polygamy hurt 19th century Mormon wives' evolutionary fitness 3Health News:High-quality care associated with lower cost in trauma 2Health News:Surviving Melanoma May Take Different Toll on Women, Men 2Health News:Surviving Melanoma May Take Different Toll on Women, Men 3
Used for retrograde pyelogram. The tip configuration occludes the ureteral orifice and stabilizes the catheter by temporarily wedging in the orifice. Supplied sterile in peel-open packages. Intended ...
... for the injection of contrast medium, anesthetic ... lumen design eliminates the need for multiple ... promote atraumatic passage into and through the ... hydrophilic polymer that when activated, attracts and ...
Used for drainage and retrograde pyelogram. Supplied sterile in peel-open packages. Intended for one-time use....
... two staggered eyes. It is made ... adapter is enclosed. They are color ... right. They are for single use, ... length is 65cm and they are ...
Medicine Products: