Rally Round Rupert has made a significant contribution of £107,000 to fund the SMA 4 Nutrition clinical trial, an important research project aimed at improving the understanding and treatment of spinal muscular atrophy (SMA) in infants and children. The trial, which began in October 2024, will be conducted over 24 months, with collaboration between Great Ormond Street Hospital in London and the University of Milan’s Department of Food Environmental and Nutritional Sciences (DeFENS).
This observational longitudinal study will focus on examining the nutritional status, body composition, energy expenditure, and dietary habits of children with SMA types I and II. The goal is to understand how these factors impact the effectiveness of the available therapeutic treatments for SMA. A key secondary aim is to evaluate the role of body composition and energy metabolism, specifically fat-free mass (kcal/kg), as potential biomarkers to predict how well patients respond to disease-modifying treatments.
The findings from this research could lead to improved personalised care for SMA patients, offering better insights into the role nutrition and metabolism play in disease progression and treatment outcomes. Rally Round Rupert’s generous donation helps make this groundbreaking trial possible, bringing hope to families affected by SMA.
Our Partners: Great Ormond Street Hospital, University of Milan, RUNFOREMMA.
