Why UK Families Shouldn’t Be Discouraged 

For families living with Rett syndrome in the UK, the recent announcement that Acadia Pharmaceuticals will not pursue UK approval for Daybue has been deeply disappointing. It’s hard not to feel deflated when a long-awaited treatment stalls before it reaches our children, especially when we know how much hope families have pinned to progress overseas. 

But it’s important to remember: this is not the end of the story — it’s just one chapter. 

The science is still moving forward 

While Daybue has paused here, gene therapy is advancing at a remarkable pace. Early trial data from both Taysha Gene Therapies and Neurogene Inc. show real, measurable gains — children and adults regaining lost skills, stabilising, and, in some cases, doing things once thought impossible. 

These results mark the first tangible proof that changing the trajectory of Rett syndrome is not a dream but an unfolding reality. 

The UK is still part of the race 

Reverse Rett has always fought to make sure that the UK stays connected to global progress. The Rett Registry UK gives us the data, the readiness and the credibility to bring trials here. Families who keep their registry records updated are part of the reason companies continue to look at the UK as a potential site for future studies. 

When the next wave of treatments — including new editing and delivery technologies funded by RSRT — reach the clinical stage, our community will be ready. 

Why your voice still matters 

If there’s one thing our community has proven since Reverse Rett began, it’s that families drive change. Every email to a GP, every shared post, every fundraiser — it all builds the momentum that gets attention, funding and action. 

For a condition as rare as Rett syndrome, progress depends on persistence. The work we do now — strengthening the registry, supporting clinical readiness, and building awareness — ensures that when new therapies arrive, no UK family is left behind. 

Hope is shifting from if to when 

We’ve come too far to lose heart now. The science is catching up to what we’ve always known: our children deserve more than management — they deserve a future. 

So yes, setbacks sting. But every study, every trial, every survey completed by a parent at midnight after a long day — they’re all steps forward on the same road. 

Together, we’ll keep walking it. 

💜 Keep hope. Stay connected. Be ready.