14/01/2026
J’adore ce message de Reverse Rett.
Il rappelle une chose essentielle : les victoires dans les maladies rares ne sont jamais isolées.
Ce qui a été possible pour la SMA nous montre ce qui devient possible pour le syndrome de Rett.
Continuons à croire, à soutenir la recherche et à refuser le “il n’y a rien à faire”.
❤️❤️ L’espoir est un moteur puissant.
You might’ve seen recent headlines about Jesy Nelson, former Little Mix star, bravely sharing that her tiny twin daughters have been diagnosed with Spinal Muscular Atrophy (SMA) Type 1, a rare genetic condition affecting muscle strength and movement.
This is a condition which, without treatment, is fatal around the age of two and Jesy is now pushing to get SMA added to newborn screening in the UK so other families can catch it early and get the treatment they need.
Here’s why this matters to us at Reverse Rett:
A few years ago, SMA was a place our Rett community knows all too well, little hope, no effective treatments, no standard newborn screening, and families left scrambling.
Today, thanks to relentless research and advocacy, game-changing therapies like Zolgensma and others are changing the outlook for many children with SMA helping them survive and reach milestones that once seemed impossible.
That kind of progress didn’t happen by accident.
It happened because people refused to accept “there’s nothing more we can do.”
And that’s exactly the mindset driving the fight against Rett syndrome.
Just like SMA, Rett once lived in a world of “there’s nothing we can do.”
But we’re now seeing gene therapies in active trials, more innovative science, and more momentum than ever and that momentum matters. Every advance in rare disease science sends ripples across the whole field.
So let’s:
Celebrate wins elsewhere in the rare disease world
Use them to fuel our vision for real breakthroughs in Rett syndrome.
Keep pushing until we’re writing about Rett just like we’re now writing about SMA 💪
Change is coming for our community 💜
Keep the hope burning 🔥
https://www.bbc.co.uk/news/articles/c1m7r840gl0o
