Comments
yourfanat wrote: I am using another tool for Oracle developers - dbForge Studio for Oracle. This IDE has lots of usefull features, among them: oracle designer, code competion and formatter, query builder, debugger, profiler, erxport/import, reports and many others. The latest version supports Oracle 12C. More information here.
Cloud Computing
Conference & Expo
November 2-4, 2009 NYC
Register Today and SAVE !..

2008 West
DIAMOND SPONSOR:
Data Direct
SOA, WOA and Cloud Computing: The New Frontier for Data Services
PLATINUM SPONSORS:
Red Hat
The Opening of Virtualization
GOLD SPONSORS:
Appsense
User Environment Management – The Third Layer of the Desktop
Cordys
Cloud Computing for Business Agility
EMC
CMIS: A Multi-Vendor Proposal for a Service-Based Content Management Interoperability Standard
Freedom OSS
Practical SOA” Max Yankelevich
Intel
Architecting an Enterprise Service Router (ESR) – A Cost-Effective Way to Scale SOA Across the Enterprise
Sensedia
Return on Assests: Bringing Visibility to your SOA Strategy
Symantec
Managing Hybrid Endpoint Environments
VMWare
Game-Changing Technology for Enterprise Clouds and Applications
Click For 2008 West
Event Webcasts

2008 West
PLATINUM SPONSORS:
Appcelerator
Get ‘Rich’ Quick: Rapid Prototyping for RIA with ZERO Server Code
Keynote Systems
Designing for and Managing Performance in the New Frontier of Rich Internet Applications
GOLD SPONSORS:
ICEsoft
How Can AJAX Improve Homeland Security?
Isomorphic
Beyond Widgets: What a RIA Platform Should Offer
Oracle
REAs: Rich Enterprise Applications
Click For 2008 Event Webcasts
In many cases, the end of the year gives you time to step back and take stock of the last 12 months. This is when many of us take a hard look at what worked and what did not, complete performance reviews, and formulate plans for the coming year. For me, it is all of those things plus a time when I u...
SYS-CON.TV
uniQure Initiates Phase I in Acute Intermittent Porphyria
AIPGENE consortium sets first step towards cure for rare disease

AMSTERDAM, December 11, 2012 /PRNewswire/ --

uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient. The study is conducted under the aegis of the AIPGENE consortium, a pan-European collaboration funded in part by the European Commission's Seventh Framework Program with the aim to develop a gene therapy for the treatment of AIP, a rare and devastating disease caused by mutations in the porphobilinogen deaminase gene (PBGD). AIP can be life-threatening and the long-term effects include irreversible nerve damage, liver cancer and kidney failure. uniQure was granted orphan drug designation for the treatment of AIP in 2009 from the European Medicines Agency.

"The start of the AIP Phase I study marks the first of four programs that will enter clinical trials over the next 12 months," says Jörn Aldag, CEO of uniQure.  "After AIP we expect clinical trials to be initiated in Parkinson's disease, hemophilia B, and Sanfilippo B. After many years of building and developing our capabilities and competencies, and the approval in November of Glybera for LPLD as the first gene therapy in the Western world, we are highly motivated to expedite the clinical development of our other advanced gene therapies."

About the AIP Phase I study

The Phase I will enroll eight patients with severe AIP at two centers: the Clinical University of Navarra, Pamplona, Spain, and the 12 de Octubre University Hospital,, Madrid, Spain. The study's primary objective is the assessment of safety and determination of the maximum tolerated dose.  Secondary objectives include tolerability of treatment, pharmacokinetics, changes in the levels of surrogate markers of activity including porphobilinogen (PBG) and delta-aminolevulinic acid (ALA), and assessment of symptom control, neuro-psychological changes and quality of life. All patients will be followed for one year, and the interim results of the Phase I are expected in Q3 2013.

About acute intermittent porphyria

Acute Intermittent porphyria (AIP) is a rare genetic disease which is caused by mutations in the porphobilinogen deaminase (PBGD) gene; one of the enzymes of the heme biosynthesis pathway.  Mutations in this gene cause insufficient activity of the protein resulting in partially disruption of heme synthesis.  This in turn leads to accumulation of toxic intermediates (ALA and PBG) giving rise to a wide variety of problems including acute, severe abdominal pains, psychiatric and neurological disorders, and muscular weakness. Acute porphyric attacks can be life-threatening and the long-term consequences include irreversible nerve damage, liver cancer and kidney failure. Currently, the only curative therapy is liver transplantation and thus, new curative options are urgently needed.  Severe AIP patients are suffering poor quality of life with palliative treatments for the different symptoms including glucose or heme infusions for metabolic replacement and inhibition of toxic metabolic production.

About AIPGENE

AIPGENE is a European Commission Framework Programme 7-funded consortium (Grant Agreement number 261506) which was put together with the aim to develop the orphan gene therapy drug AAV5-AAT-PBGD (AMT-021) for the treatment of Acute Intermittent porphyria (AIP). The consortium's objective is to contribute to alleviating the negative impact of this disease on the quality of life of the patients and their families. Overall coordinator of the project is the Centre for Applied Medical Research (CIMA) at the University of Navarra, Pamplona, Spain. Apart from uniQure, other members of the consortium are the Clinical University of Navarra, Pamplona, Spain; Karolinska University Hospital, Stockholm, Sweden; German Cancer Research Center (NCT-DKFZ), Heidelberg, Germany; DIGNA Biotech, Pamplona, Spain; Servicio Madrileno de Salud, Madrid, Spain.

About uniQure

uniQure is a world leader in the development of human gene based therapies. uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure's product pipeline of gene therapy products in development comprise hemophilia B, acute intermittent porphyria, Parkinson's disease and Sanfilippo B. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene, and allows uniQure to pursue its strategy of focusing on this sector of the industry. uniQure's largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at http://www.uniqure.com.

SOURCE uniQure B.V.

About PR Newswire
Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

SOA World Latest Stories
SYS-CON Events announced today Isomorphic Software, the global leader in high-end, web-based business applications, will exhibit at SYS-CON's DevOps Summit 2015 New York, which will take place on June 9-11, 2015, at the Javits Center in New York City, NY. Isomorphic Software is the gl...
ScriptRock makes GuardRail, a DevOps-ready platform for configuration monitoring. Realizing we were spending way too much time digging up, cataloguing, and tracking machine configurations, we began writing our own scripts and tools to handle what is normally an enormous chore. Then...
SYS-CON Events announced today that Cloudian, Inc., the leading provider of hybrid cloud storage solutions, will exhibit at SYS-CON's 16th International Cloud Expo®, which will take place on June 9-11, 2015, at the Javits Center in New York City, NY. Cloudian, Inc., is a Foster City, ...
The BPM world is going through some evolution or changes where traditional business process management solutions really have nowhere to go in terms of development of the road map. In this demo at 15th Cloud Expo, Kyle Hansen, Director of Professional Services at AgilePoint, shows Agile...
Verizon Enterprise Solutions is simplifying the cloud-purchasing experience for its clients, with the launch of Verizon Cloud Marketplace, a key foundational component of the company's robust ecosystem of enterprise-class technologies. The online storefront will initially feature pre-b...
"People are a lot more knowledgeable about APIs now. There are two types of people who work with APIs - IT people who want to use APIs for something internal and the product managers who want to do something outside APIs for people to connect to them," explained Roberto Medrano, Execut...
Subscribe to the World's Most Powerful Newsletters
Subscribe to Our Rss Feeds & Get Your SYS-CON News Live!
Click to Add our RSS Feeds to the Service of Your Choice:
Google Reader or Homepage Add to My Yahoo! Subscribe with Bloglines Subscribe in NewsGator Online
myFeedster Add to My AOL Subscribe in Rojo Add 'Hugg' to Newsburst from CNET News.com Kinja Digest View Additional SYS-CON Feeds
Publish Your Article! Please send it to editorial(at)sys-con.com!

Advertise on this site! Contact advertising(at)sys-con.com! 201 802-3021


SYS-CON Featured Whitepapers
ADS BY GOOGLE