As we continue our ‘Every Piece of the Puzzle’ Campaign for SMA Awareness Month, we want to say a heartfelt thank you to every member of the SMA community who has shared their story over the years, raised their voices and helped drive change.

Every story, conversation and voice has helped build greater understanding of SMA and shine a light on the realities of living with the condition. Whether through advocacy, awareness raising or simply sharing your experiences, your contributions have helped bring people together and show why every piece of the puzzle matters.

This month, we’re celebrating the strength and determination of the SMA community and the collective impact we can make when we come together.

Thank you for being part of the puzzle and helping shape what comes next.

Gary Edgecombe 

As part of SMA UK’s ‘All Pieces of the Puzzle’ campaign for SMA Awareness Month, I’m proud to be talking about one of the pieces that means so much to me: community. Living with SMA Type 3 myself, I know how important it is to feel connected to people who understand the challenges, celebrate the milestones and simply get it. Since joining SMA UK in 2023, I’ve had the privilege of meeting so many members of our community through events, conversations and our Living With SMA podcast. Every connection helps remind us that none of us has to navigate life with SMA alone. 

Our community is made up of so many different people, experiences and voices, but together we are stronger. Whether you’ve been part of the SMA community for years, are newly diagnosed, support someone with SMA, or are simply learning more about the condition, you are an important piece of the puzzle. This Awareness Month, I hope we can celebrate the strength, support and shared experiences that bring us together, because our community really does make a difference. 

Ben Morris  

Hello! For those of you who don’t know me, my name is Ben, I’m 25, and I have Spinal Muscular Atrophy Type II. I was diagnosed at the age of 11 months, and have essentially required 24 hour care ever since. I’m a full-time electric wheelchair user, require ventilation overnight, and have a full-time job just to make things even more interesting. 

Raising awareness of SMA, is something that I have done for years. From talking to a room full of scientists in Switzerland, to answering boring surveys about how much I can breathe… or not, I have done it all!  

In a time where the condition is under the spotlight more than ever, it is so important to show every angle of those impacted by the disease. The community knows that just because someone has SMA, it doesn’t mean that it’s all doom and gloom. Yes, it can be devastating for some families but by raising awareness of SMA, we can highlight the achievements that people make, even with the challenges they face every day. I would like to think that by doing this, it will give hope to future families diagnosed with SMA.  

I’ve been trying to raise awareness for the condition, for as long as I can remember. This mainly stems from my parents, making sure that I looked on the brighter side of things when I was a child. From a personal standpoint, giving talks and raising awareness of the condition, was my way of getting my head around everything that was going on. 

I would ask anyone and everyone to raise as much awareness as possible, as it doesn’t only have an impact on the general public, it has an impact on you as well.” 

Kacper Rucinski

“In 2016, at the World Muscle Society Congress in Granada, I met Dr Laurent Servais and began exploring whether children from across Europe could access the newly announced drug nusinersen at his clinic in Paris. Soon, thanks to his ingenuity, tens of families began travelling to Paris for treatment, receiving the life-saving drug free of charge through the Expanded Access Programme. For many families, including ours, Paris became a place of hope and the beginning of friendships and collaborations that would shape the SMA community for years to come.

Back in the UK, however, access proved much harder. NHS England initially refused to allow the programme, and parents and SMA organisations, including TreatSMA that I helped to found, began campaigning for change. It took months of discussions, campaigning and behind-the-scenes negotiations for the NHS England to eventually agree.

Securing long-term NHS access was a much harder battle as we faced competing motives. We had to prevent the manufacturer from going the easier way that would limit the treatment only to around a hundred children with the most severe symptoms. We needed NICE to be flexible with its cost-effectiveness thresholds. With the help of our high-placed contacts and the amazing clinical community, NICE genuinely engaged in the process, doing everything it could to find a way of recommending this very expensive drug for as many patients as possible. However, it took months of discussions, pharmacoeconomic modelling, campaigning, and, ultimately, protests outside Parliament and NICE offices before the tide began to turn.

On 14 May 2019, NICE announced that nusinersen would be made available on the NHS for the majority of people living with SMA. We continued to push for even wider access, eventually formally appealing the proposed recommendation. A few weeks later, NICE agreed to extend access to more children with milder symptoms. It was not everything we had hoped for, but after years of persistence by families, clinicians and SMA organisations, the treatment era had finally begun.”