MECP2 Duplication Syndrome is caused by the same gene as Rett Syndrome. In Rett Syndrome, there are mutations or deletions in the MECP2 gene, whereas in MECP2 Duplication Syndrome, the gene is mistakenly duplicated. The syndrome has been diagnosed mostly in boys.

At Reverse Rett we have a dedicated Cure MECP2 Duplication UK fund which is supported by a small group of committed families and their friends and connections.

IONIS Pharmaceuticals is planning to conduct first-in-patient human trials in 2024 (ION 44) with a potential cure. However, funding is crucial to ensure the success of these trials. There are also other gene therapies waiting for funding to expedite their development.

We need your help to make this happen. We need a cure and we need it fast! With your support and contributions to the MECP2 Duplication Fund, we can bring hope to families like ours and change the lives of children affected by this awful disease.