Rett Syndrome is a life shattering brain disorder affecting young and usually apparently healthy children, leaving them with medical complexities and severe disabilities for life. There are no approved treatments. Parents have to watch as their child deteriorates before their eyes; a progression which can last their whole childhood.

I know it well. My own daughter, Amber, and step-daughter, Beth, both have Rett Syndrome. Both were happy and typical toddlers. Both now need 24 hour nursing care. Amber's needs are so high, we can no longer safely manage her at home; a situation which I have found excruciating, however necessary, however good her care home is, however close by we are and however often we are with her. It just isn't what anyone wants for their child.

We are getting to a point with Rett Syndrome though, where it is clear that in the near future, we are likely to have a gene therapy that works. Gene therapy will target the underlying cause of the condition and should be able to correct the problem at its root.

What this will mean for children, teenagers and adults already living with Rett Syndrome remains to be seen. 

We hope it will mean real improvements in their health; that they are able to live longer and will be less at risk from serious illness and death.

We hope it will mean improvements in their abilities, that they will be able to do some of the many things they now aren't able to do, like regain some speech, move more freely in their bodies, perhaps regain some of the use of their hands. Any improvement in mobility would be so welcome. When you have this level of disability, the difference between even being able to bear weight for a few seconds and not, makes a tremendous difference on a practical level and for your health.

But gene therapies for young children, those whose Rett symptoms have not fully taken a hold, is likely to have a profound effect on their lives and futures. Children who are able to access gene therapies at a young age will undoubtedly live, very different lives to our girls who have endured so much.

Any donation means the world to me, Andy and all the parents and families of affected children, young people and adults with Rett Syndrome. Thank you.

About Reverse Rett

Reverse Rett is a patient advocacy and research organisation working to bring these and other treatments for Rett Syndrome to the UK. We believe that everyone with Rett deserves the opportunity to access these emerging therapies. Reverse Rett work to identify and register every affected person, to recruit patients for UK clinical trials, fund the UK clinical research and treatment Centre, (CIPP Rett) and provide ongoing funding for further lab research seeking to refine gene therapy techniques via the US based RSRT. 

Read more here: reverserett.org.uk

For Rett Registry UK: https://www.rettregistry.uk/