Reverse Rett

Cure MECP2 Duplication UK

The Cure MECP2 Duplication UK Fund at Reverse Rett supports the efforts of MECP2 Duplication families in Europe working to speed treatments and a cure for their children. Same gene, same goal!
£216,476
raised of £300,000 target
by 9712 supporters
RCN 1136809

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Story

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.

About the charity

Reverse Rett

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RCN 1136809
Reverse Rett is a patient advocacy and research organisation focused on delivering treatment for Rett Syndrome to all affected. Read more here: reverserett.org.uk For Rett Registry UK: https://www.rettregistry.uk/

Donation summary

Total raised
£216,475.80
+ £22,438.38 Gift Aid
Online donations
£212,067.46
Offline donations
£0.00
Direct donations
£8,539.84
Donations via fundraisers
£207,935.96

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