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:4/29/2016)... ... 29, 2016 , ... In an article published April 16th on ... Botox and lip injections, which she underwent in order to feel more at home ... Arts Festival. The article explains that Ms. Mirmelli’s situation is not unique; many plastic ...
(Date:4/29/2016)... , ... April 29, 2016 , ... On Tuesday, April ... across the Southeast, celebrated the signature of Gov. Nathan Deal on SB 258, the ... Duncan (R - Cumming), offers a 70% tax credit to individuals and corporations which ...
(Date:4/29/2016)... , ... April 29, 2016 , ... Coast Dental Fort ... 2016, at its new location in the Exchange Furniture Mall at 112 Vilseck Road ... a 50-inch Samsung Smart TV. Plus attendees will have the opportunity to meet general ...
(Date:4/29/2016)... , ... April 29, 2016 , ... ... of patented products, announces the Gyrociser, an exercise invention which aids in proper ... billion," says Scott Cooper, CEO and Creative Director of World Patent Marketing. "Globalization ...
(Date:4/29/2016)... ... April 29, 2016 , ... Reltok Nasal Products proudly announces that Boston ... and neck/ear, nose and throat specialty, has added the KOTLER NASAL AIRWAY™ to its ... is a newly patented safety device secured by nasal surgeons onto the floor ...
Breaking Medicine News(10 mins):
(Date:4/27/2016)... Tie-up with Government hospitals as ... to save newborns   Fortis La Femme, ... collaboration with Breast Milk Foundation (BMF), a non-profit organization within ... Bank, ,Amaara, in Delhi-NCR today. This non-profit centre recognizes that ... and should be available to babies deprived of mother,s milk.  ...
(Date:4/27/2016)... Shire plc (LSE: SHP, NASDAQ: ... Financial Officer, will present at the Deutsche Bank 41st Annual ... on Wednesday, May 04, 2016, 10:00 am EDT (15:00 BST). ... the Presentations and Webcasts section of Shire,s Investor website at ... be available on this same website for approximately 90 days. ...
(Date:4/27/2016)... , April 27, 2016 ... ), a developer of a new generation of ... positive overall survival results for Paclical/Apealea in the ... 789 patients with epithelial ovarian cancer. These preliminary ... Paclical/Apealea in combination with carboplatin versus Taxol in ...
Breaking Medicine Technology: