Navigation Links
Settings standards for research into Rett syndrome
Date:10/31/2012

There is an urgent need for new drugs to treat Rett syndrome, a rare and severe neurological disease mainly affecting girls. A bottleneck in drug development for this syndrome is a lack of clarity at the level of preclinical research. Key researchers in this field now tackle this issue, proposing standards and guidelines for Rett syndrome research, in an Open Access review article published on Oct. 31, 2012 in Disease Models & Mechanisms (DMM) at http://dmm.biologists.org/. This "state of the science" assessment serves as a comprehensive resource of all findings and citations related to Rett syndrome.

Rett syndrome is a rare neurological disease that affects girls almost exclusively. Affected children appear to grow and develop normally for the first 6-12 months of life, but then begin to show symptoms including slow development, problems crawling or walking, and diminished eye contact. As the syndrome progresses, affected children show a regression of many skills, with loss of purposeful hand use, speech and basic motor function. Severe cases can experience problems with heart, respiratory and gastrointestinal function. Rett syndrome is extremely difficult on affected girls and their families. Although the disease-causing gene (called MECP2) has been identified, there is no cure, and only limited symptomatic treatments are available. A key to developing new treatments for this devastating disorder is to improve systems and standards at the preclinical research level.

The new review article reports on outcomes from a workshop held in September of 2011 focused on the state of the art in animal studies of Rett syndrome. The workshop was convened by important funders of Rett syndrome research, including the National Institute of Neurological Disorders and Stroke (NINDS), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the International Rett Syndrome Foundation (IRSF) and the Rett Syndrome Research Trust (RSRT). Workshop participants included key members of the Rett syndrome research community, including basic scientists, clinicians, and representatives from the National Institutes of Health (NIH), the Food and Drug Administration (FDA), the pharmaceutical industry and private foundations. Several workshop participants contributed to the article, and the corresponding authors are Dr. David Katz (Case Western Reserve University School of Medicine), Dr. Laura Mamounas (National Institute of Neurological Disorders and Stroke) and Dr. Huda Zoghbi (Baylor College of Medicine).

The main aim of the workshop was to identify crucial knowledge gaps in Rett syndrome research at the preclinical level, and to suggest scientific priorities and best practices for the use of animal models in preclinical evaluation of potential new therapeutics. As explained in the new review article, the combination of an urgent need for effective treatments for Rett syndrome, coupled with the availability of good mouse models, is a driving force for studies that can identify and test new drugs. The outcome of the workshop included a set of recommended guidelines for animal studies of Rett syndrome, to ensure that decisions to initiate costly clinical trials will be founded on reliable data that is produced using standardized study design and transparent reporting. Ultimately, the increased level of rigor in animal studies should shorten the time to effective treatments.


'/>"/>
Contact: Sarah E. Allan, PhD
sarah.allan@biologists.com
International Rett Syndrome Foundation
Source:Eurekalert

Related medicine news :

1. Magnets in iPad2 May Alter Settings on Brain Shunt Devices: Study
2. Gold standards of success defined for AF ablation
3. Alcohol advertising standards violations most common in magazines with youthful audiences
4. Local Woodinville Contractor, Washington State Kitchen and Bath, Combats Toxic Mold, Adhering to the Toughest Mold Protection Standards in the Nation with New Promotion
5. New standards for treating traumatic shoulder injuries to improve patient care
6. Job injuries among youth prompt calls for better safety standards
7. Attention, parents: UC research reveals a secret about your medicine cabinet
8. Nova Scotia research team proves peer pressure can be used for good
9. Mechanism found for destruction of key allergy-inducing complexes, Stanford researchers say
10. Feinstein Institute researchers discover that bean used in Chinese food could protect against sepsis
11. AACR, Komen for the Cure honor researcher with lecture on science of cancer health disparities
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/27/2016)... ... June 27, 2016 , ... ... payment industry today announced its strategic partnership with Connance, a healthcare industry ... The two companies’ proven, proprietary technology combine to provide health systems, hospitals ...
(Date:6/26/2016)... ... June 26, 2016 , ... On June 10-11, 2016, A ... 2016 Cereal Festival and World’s Longest Breakfast Table in Battle Creek, MI, where the ... history as home to some of the world’s leading providers of cereal and other ...
(Date:6/26/2016)... Orion, Clarkston, Michigan (PRWEB) , ... June 26, ... ... with respect to fertility once they have been diagnosed with endometriosis. These women ... intercourse but they also require a comprehensive approach that can help for preservation ...
(Date:6/25/2016)... Viejo, California (PRWEB) , ... June 25, 2016 , ... ... to fit their specific project," said Christina Austin - CEO of Pixel Film Studios. ... fully customizable and all within Final Cut Pro X . Simply select a ...
(Date:6/25/2016)... (PRWEB) , ... June 25, 2016 , ... On Friday, ... presented a Bronze Wellness at Work award to iHire in recognition of their exemplary ... part of the 7th annual Maryland Workplace Health & Wellness Symposium at the BWI ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... Va. , June 24, 2016 The ... set of recommendations that would allow biopharmaceutical ... (HCEI) with entities that make formulary and coverage decisions, ... the "value" of new medicines. The recommendations ... does not appear on the drug label, a prohibition ...
(Date:6/24/2016)... Mass. , June 24, 2016   Pulmatrix, ... pharmaceutical company developing innovative inhaled drugs, announced today that ... Russell Investments reconstituted its comprehensive set of ... "This is an important milestone for Pulmatrix," ... will increase shareholder awareness of our progress in developing ...
(Date:6/23/2016)... Calif. , June 23, 2016 Any dentist ... many challenges of the current process. Many of them do ... of the technical difficulties and high laboratory costs involved. And ... to offer it at such a high cost that the ... it. Dr. Parsa Zadeh , founder of ...
Breaking Medicine Technology: