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100% of money raised funds clinical research projects
Rally Round Rupert is a Registered Charity - 1201716
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About The Charity

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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:


a. Working to improve the understanding and knowledge of Spinal Muscular Atrophy (SMA) among the public.


b. Providing financial support to further advance clinical research into Spinal Muscular Atrophy (SMA).

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What is SMA?

Spinal Muscular Atrophy (SMA) is a genetic, neuromuscular condition that causes muscle weakness and wastage due to the loss of motor neurons. Each year about 1 in 10,000 children are born with the condition and typical symptoms of SMA type 1 (the most severe form of the condition) are:

• Floppy or weak neck, arms, and legs (hypotonia)
• Movement problems – such as difficulty sitting up, crawling, or walking
• Swallowing difficulties
• Breathing difficulties
• Twitching or shaking muscles (fasciculations / tremors)
• Bone or joint problems – such as curvature of the spine (scoliosis and kyphosis)


Before 2016, without intervention and treatment, most children with SMA type 1 were expected to live for less than two years. Today however, early diagnosis and immediate medical intervention can significantly abate the impacts of SMA, drastically improving the quality of life for children affected, however very few people, including clinicians know about the disease.
 

As these new treatments are now more widely available, there is a whole new community of children that are surviving the condition. These children present unique lifelong disablement and clinicians now need to develop new protocols and treatments to care for these children, that would have otherwise died.

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100% of money raised funds clinical research projects
Rally Round Rupert is a Registered Charity - 1201716
Charity commission logo

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