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Gene therapy trials for Rett syndrome are now underway. But the biggest battle for our community lies ahead; getting these life-changing treatments recommended for NHS funding. Your donation will help us build the strong evidence package we need to drive these new treatments into the homes and live
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Beneficiaries
Situation
Rett Syndrome is a progressive, life-threatening brain disorder which strikes toddlers between the ages of 1-2, leaving them with severe disabilities and medical problems for life. There are currently no approved treatments but gene therapy trials are now underway. These treatments will be expensive. Reverse Rett is a small but national patient advocacy organisation that will need to be prepared to demonstrate UK burden of disease and need for treatments so they are recommended for funding.
Solution
Reverse Rett will commission an independent specialist medical consultancy to work with us to develop an action plan to build the tools and resources needed for access to these potentially life-changing medicines for our community. We believe that everyone with Rett Syndrome, regardless of age or gender deserves equal access to emerging treatments. This comprehensive work will help us effectively demonstrate the need for and benefits of treatment across age groups and disease stages.
