Fighting to Reverse Rett
In 2012 Bone Marrow Transplants were shown to arrest symptoms of Rett Syndrome in a mouse model. Further research is underway to assess the potential for clinical trials. This project will help drive this work out of the lab and into the lives of girls and women living with Rett Syndrome today.
January 2013 - January 2014
Rett Syndrome is a complex post natal neurological disorder. The disease robs previously healthy female toddlers of basic life skills, including speech, mobility and hand use, leaving them multiply disabled and medicallly compromised for life. There is no cure. But this year, researchers have shown bone marrow transplants to be a viable potential therapeutic for the condition. Much work needs to be done to shift these results from the lab into the homes and lives of girls and women with Rett.
Last year’s BG Christmas Challenge enabled families of daughters with Rett to have a direct impact on the potential for treatment for this devastating condition by fundraising to support the project entitled ‘Immune modulation as a new therapeutic approach for Rett Syndrome,’ which yielded results demonstrating the arrest of symptoms in a mouse model. Work is now underway to prepare for clinical trials. Funds are needed to ensure that these clinical trials can get underway.
To deliver £80K over 2 quarters to support further work on preparation for and initiation of CT.
Activities» We will recruit as many fundraisers as we can to raise the funds to meet our target of £40,000 in online donations.
Success will be...the delivery of these funds to the research project/medical establishment implementing clinical trials.
Rett Syndrome is a devastating condition for which there is currently no treatment. Any potential therapeutic options provide hope for families of girls and women battling this condition today. We hope that our support of these initial clinical trials will enable us to further explore the potential of bone marrow transplants as an option for treating girls and women with Rett and hopefully ameliorating some of the many complex symptoms they endure throughout their daily lives.
We ensure the scientific validation and medical efficacy of the research projects we support through the shared use of the Scientific Advisory Board of the Rett Syndrome Research Trust, which includes many key contributors to the field of Rett research. All research proposals undergo a rigorous peer-review process before being recommended for funding by Rett Syndrome Research Trust UK.
We will report to donors in the following ways:
1. Updates to News blog on Rett Syndrome Research Trust UK website.
2. Alerts for all of these updates will be shared through social networking, including Twitter/ facebook page etc.
3. Monthly newsletter
Budget - Project Cost: £80,000Loading graph....
Amount Heading Description £80,000 Support for CT Funding to support further research into viability of CT and subsequent initiation of those trials.
Rett Syndrome Research Trust UK is a national organisation with one goal; to speed treatment for Rett Syndrome. Our registered office is based in Buckinghamshire. For this project we will be utilising technology to involve families and donors from all across the UK.
The ulitimate beneficiaries of our project will be girls and women with Rett, their families and communities. Rett Syndrome affects approximately 1:10,000, similar numbers to Cystic Fibrosis, yet it is largely unrecognised. This project will bring much needed awareness of this devastating condition. It will empower families, giving them a way to impact their own daughter’s future as they help us explore the possibility of BMT as a potential treatment through the implementation of clinical trials
Rett Syndrome Research Trust UK is the only UK charity exclusively focused on the development of treatments and cures for Rett Syndrome. We know that our ability to ensure the delivery of treatments and cures directly correlates to the quality, efficiency and focus of the work we do. Winners of Best New Charity at both the Charity Times Awards 2011 and Just Giving Awards 2012, our charity has been nationally recognised for achieving our goals.
Read more about the Charity running this project.
Rachael Bloom will oversee the co-ordination of fundraising, pledges and the overall campaign to ensure that donations are made on Challenge Day.
As Community Fundraising Manager, Andy will support fundraisers in the run up to the Challenge and will ensure that donations are made on time.
Helen will engage with fundraisers, providing them with information and support as pertains to the Challenge in preparation for the Big Day!
Kelly will provide as needed support to fundraisers by phone, email or social media, as pertains to the specifics of their fundraising efforts.