Why fundraising for Reverse Rett is still Vital
2024 is already proving to be a promising year for the UK Rett Community with two Rett syndrome gene therapy trials approved back in early January.
But until the trials are able to demonstrate that these new treatments are safe, effective and ultimately recommended for funding by NICE/SMC etc., our work at Reverse Rett continues. Before we can even get to that point, there are some key things that we need to do:
Find missing patients: We need to find and register at least 70% of the estimated UK Rett population on the Rett Registry UK. This is because NICE/SMC want to see that the actual patient population marries up with the disease incidence.
Getting ready for UK gene therapy clinical trials: Much work goes on to help facilitate the implementation of these UK trials including building patient voice regulatory contributions and making films and providing presentations to companies working in this space.
Building the case for access to treatments: We need to build tools and resources to support the campaign for UK regulators and payers to recommend funding for these treatments when they are proven to work so that there is minimum delay to the wider population accessing emerging treatments as and when they are proven to be safe and effective.
Development of the UK Rett syndrome clinical network: We’re working to help develop a cohesive network of trial sites and clinical teams to deliver emerging treatments once they are demonstrated to be safe and effective through clinical trials.
Bridging critical gaps in clinical care: We continue to fund the CIPP Rett Centre, the only UK treatment centre dedicated to managing neurobehavioral health in Rett syndrome in the UK. The CIPP Rett team will be vital in making the most of clinical outcomes in older and or more complex patients with Rett syndrome, post access to emerging disease modifying treatments and critical now, in helping patients stay healthy until these treatments become accessible to them.
We need to continue to share the latest information and resources about staying healthy with Rett syndrome to ensure that as many of our children, young people and adults with Rett as possible are here to benefit from these emerging disease modifying treatments as they become available.
At Reverse Rett, we are a small team but we achieve a lot with the funds we raise. However, unlike some other rare disease charities, we have to raise more than our running costs so that we are able to invest in programs like the CIPP Rett Centre, the Rett Registry UK, our outreach work and ongoing lab research via RSRT.
We do not receive any government funding and we do not have a dedicated Trusts and Foundations Fundraiser. Almost all of our funds come from families, their friends and connections holding local events and taking on personal and team challenges or asking for birthday donations from Facebook.
It’s a very positive time for the Rett community both here in the UK and around the world but we still need your help, arguably more than ever before.
Got a brilliant fundraising idea but need a little help to make it happen? Drop us a email at [email protected], we’re available to provide guidance and support to help you bring your idea to fruition.