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Raising awareness and funds in support of clinical research for spinal muscular atrophy (SMA)

Raising awareness and funds in support of clinical
research for spinal muscular atrophy (SMA)

100% of money raised funds clinical research projects.

Our Objectives

To relieve the need and protect the health of children and their families affected by Spinal Muscular Atrophy (SMA) throughout England & Wales for the public benefit by:

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Amount Raised
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Clinical Trials Funded
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Making a Difference: Where Your Donations Go

Rally Round Rupert has been making significant strides in its mission. rough a combination of generous donations and fundraising efforts, we have funded clinical projects that will dramatically improve the lives of those with SMA.

Additional Allocations

Key Allocations

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.

Trial to commence October 2024, duration 24 months.

Our Partners:

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.

Trial commenced June 2024. Duration 24 months.

Our Partners:

News and Updates