Navigation Links
IRSF announces funding of first clinical trial with disease-modifying therapy for Rett syndrome
Date:9/21/2009

(Cincinnati, OH) - Today, the International Rett Syndrome Foundation announced that it will provide $200,000 in 2009 to support a newly proposed clinical trial with a growth-factor based treatment for Rett Syndrome. The study will be carried out by a team of clinical trials specialists at the Boston Children's Hospital, led by Dr. Omar Khwaja MD, PhD and will be the first potentially disease modifying therapy ever to be tested in Rett syndrome patients. Investigators will test a drug called Increlex. This is an engineered form of the human protein Insulin-like Growth Factor-1, an FDA approved drug for children with short stature due to severe Primary IGF-1 deficiency. Preliminary evidence in animal models of Rett syndrome has recently suggested that targeting the IGF-1 signaling axis may provide a potential avenue for therapy.

"We are pleased to announce the funding of this groundbreaking new clinical study which presents a novel therapeutic strategy for the treatment of Rett syndrome" said Dr. Antony Horton Chief Scientific Officer at the International Rett Syndrome Foundation. "This study will be carried out by a collaborative network of leading clinicians and neuroscientists in some of the World's most prestigious research institutes at the cutting edge of biomedical research. Through our funding of this study we demonstrate our commitment to moving forward the most advanced treatments to prevent, treat and ultimately reverse Rett syndrome".

Dr. Omar Khwaja, principal investigator on the study commented further "Since the 2007 studies which demonstrated potential reversal of Rett syndrome in animal models, there has been a paradigm shift in our thinking about ways to treat this devastating condition. Our pilot study will utilize an existing FDA approved medication, seeking to boost growth of synapses in the brain of patients with Rett syndrome".

Increlex will be provided gratis by Tercica Inc., A Subsidiary of the IPSEN Group, who have reviewed and approved the trial through their internal scientific review board.

Dr. Khwaja and his team have also secured additional funding for this clinical trial through a grant provided by Harvard University's Catalyst Pilot Awards for Clinical Translational Research


'/>"/>

Contact: Mary Joyce Griffin
mgriffin@rettsyndrome.org
513-870-4378
International Rett Syndrome Foundation
Source:Eurekalert

Related biology news :

1. Selexis Announces Advanced Approach to Maximize Power of Genetic Elements for Rapid Development of High Performance Cell Lines
2. LaserCard Corporation Announces Expansion of Middle East Project
3. BIO-key(R) Announces Contracts for New Criminal Justice Data Sharing Solution
4. Balzan Foundation announces 2007 winners
5. The American Society of Plant Biologists announces 2007 awards
6. ASPB announces Summer Undergraduate Research Fellowship 2007 recipients
7. NIST announces 56 new awards for innovative technology research and development
8. Titanium Group Announces Contract With Haitong Securities
9. Measurement Specialties, Inc. Announces Impact of German Business Tax Reform 2008 Resulting in Lower Combined German Tax Rate - Impacts 2Q08 Reported Financial Results
10. FIJI Water announces sustainable growth initiative with commitment to help mitigate climate change
11. HGS announces positive Phase 2 LymphoStat-B at ACR meeting
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/20/2017)... , March 20, 2017 PMD Healthcare announces ... spirometer and Wellness Management System (WMS), a remote, real-time ... in 2010, PMD Healthcare is a Medical Device, Digital ... mission dedicated to creating innovative solutions that empower people ... that intent focus, PMD developed the first ever personal ...
(Date:3/9/2017)... and MOUNTAIN VIEW, Calif. , ... Well Made Simple," and 23andMe , the leading ... better food choices.  Zipongo can now provide customers with ... preferences, health goals and biometrics, but also genetic markers ... choices. Zipongo,s personalized food decision support platform ...
(Date:3/2/2017)... Summary This report provides all the ... interests and activities since 2010. ... Read the full report: http://www.reportlinker.com/p03605615-summary/view-report.html ... report provides an in-depth insight into the partnering activity of ... On demand company reports are prepared upon purchase to ensure ...
Breaking Biology News(10 mins):
(Date:3/23/2017)... Calif., March 23, 2017  BioPharmX Corporation (NYSE ... for the dermatology market, today reported financial results ... 2017, and will provide an update on the ... year. "We are pleased to report ... for BioPharmX," said President Anja Krammer. "We achieved ...
(Date:3/23/2017)... VILLAGE, Colo. , March 23, 2017  Agriculture ... in Series A financing and note conversion to commercialize ... Cool Planet is focused on developing products that are ... nearly $30 million in the last 18 months. This ... and North Bridge Venture Partners. The ...
(Date:3/22/2017)...  Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical ... address significant unmet medical needs in rare diseases, ... ended December 31, 2016. "2016 ... we broadened our pipeline and pursued our vision ... with an initial focus on endocrinology," said ...
(Date:3/22/2017)... 2017 The global chronic kidney ... report by Transparency Market Research (TMR). The top four ... AbbVie Inc., accounted for a share of only 41.4% ... this market are focusing aggressively on mergers, acquisitions, and ... likely to lead to market consolidation in the next ...
Breaking Biology Technology: