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:6/23/2017)... N.Y. and ITHACA, N.Y. ... ) and Cornell University, a leader in dairy research, ... with bioinformatics designed to help reduce the chances that ... With the onset of this dairy project, Cornell University ... Consortium for Sequencing the Food Supply Chain, a food ...
(Date:5/16/2017)... --  Bridge Patient Portal , an enterprise patient ... Systems , an electronic medical record solutions developer ... a partnership to build an interface between the ... products, including Centricity Practice Solution (CPS), Centricity Business ... integrations will allow healthcare delivery networks using GE ...
(Date:4/18/2017)... Inc., a global expert in SoC-based imaging and computing solutions, has ... features the company,s hybrid codec technology. A demonstration utilizing TeraFaces ® ... be showcased during the upcoming Medtec Japan at Tokyo Big Sight ... Las Vegas Convention Center April 24-27. ... Click here for an image of the ...
Breaking Biology News(10 mins):
(Date:10/9/2017)... 2017  BioTech Holdings announced today identification and ... ProCell stem cell therapy prevents limb loss in ... demonstrated that treatment with ProCell resulted in more ... compared to standard bone marrow stem cell administration.  ... reduction of therapeutic effect.  ...
(Date:10/9/2017)... ... October 09, 2017 , ... The Giving Tree Wellness ... targeting the needs of consumers who are incorporating medical marijuana into their wellness ... Arizona. , As operators of two successful Valley dispensaries, The Giving Tree’s two ...
(Date:10/7/2017)... ... October 06, 2017 , ... ... its ProxiMeta™ Hi-C metagenome deconvolution product, featuring the first commercially available Hi-C ... software to perform Hi-C metagenome deconvolution using their own facilities, supplementing the ...
(Date:10/6/2017)... ... October 06, 2017 , ... On Tuesday, October 24th, ABC² ... the first-ever adaptive clinical trial for glioblastoma (GBM). The featured speaker will be ... and open to the public, but registration is required. , WHAT: ABC² ...
Breaking Biology Technology: