We work to raise understanding of SMA across the UK, empowering families with information and support.
Our funding prioritises clinical studies and research projects that move the needle in SMA care and treatment.
We commit to full transparency by openly sharing reports, impact data, and how funds are allocated.
This observational longitudinal study aims to investigate nutritional status, body composition, energy expenditure and dietary habits in infants and children with SMA types I and II treated with the available therapeutic options. e secondary aim is to evaluate the role of body composition and energy metabolism of fat free mass (kcal/kg) as biomarkers to predict response in patients receiving disease-modifying treatments.
By improving knowledge around nutrition and metabolism, this study supports better long-term outcomes for children with SMA.
Our Partners
Great Ormond Street Hospital - LONDON
Department of Food Environmental and Nutritional Sciences (DeFENS)
University of Milan
RUNFOREMMA
A Monocentric, Prospective and Longitudinal study investigating the acceptability, feasibility, safety and efficacy of an optimised rehabilitation program for treated patients with SMA compared to the current rehabilitation program in the United Kingdom.
By improving knowledge around nutrition and metabolism, this study supports better long-term outcomes for children with SMA.
Our Partners
University of Oxford
ACE SMA (co-funding the study
2025
2024 was a year of progress, passion, and profound impact. As we reflect on the past twelve months, we’re proud to share the milestones captured in our RRR Annual Report 2025 —a testament to what can be achieved when a community comes together with a shared mission: to support families affected by Spinal Muscular Atrophy (SMA) and accelerate clinical research that changes lives.
Your donation helps fund vital research into Spinal Muscular Atrophy (SMA), bringing hope to children, adults, and families affected by this rare condition.
